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Issue 921 (09 October 2017)


Welcome to BioNews by email, published by the Progress Educational Trust, providing you with news, comment and reviews on genetics, assisted conception, embryo/stem cell research and related areas.

Visit the BioNews website at where you can subscribe for free to receive BioNews by email in one of three formats, and search the archive of more than 6,000 articles.





News Digest




Making dreams reality

09 October 2017

By Sarah Norcross

Page URL: Appeared in BioNews 921

There was lot of commentary about UK Prime Minister Theresa May's speech last week. The coughing, the prankster, the Frida Kahlo bracelet, bits of the slogan behind her falling off, and of course the 'British dream'.

Not many people picked up on one thing the Prime Minister said: 'It has always been a great sadness for me and Philip that we were never blessed with children. It seems some things in life are just never meant to be.' A rather fatalistic comment.

A lot of infertile people who live in England are seeing their prospects of state-funded fertility treatment cut. With those cuts some will see their dream of having a family vanish, while others will spend many thousands of pounds on private treatment. Are these couples supposed to accept their lot and not demand that the NICE Guideline recommending three full cycles of fertility treatment be implemented in England, Wales and Northern Ireland, as it is in Scotland? Should they just resign themselves to the fact that an unelected Clinical Commissioning Group in Cambridge or Croydon (see BioNews 917 and 893) can consult on cutting funding for IVF, ignore the response of the local people, and cut the service?

This is an issue which BioNews has been covering for years, and which the Progress Educational Trust (PET, the charity which publishes BioNews) has been campaigning about for years, because PET seeks to improve choices for people affected by infertility.

Meanwhile this week, a less expensive method fertility treatment has been launched in London's Harley Street of all places. Certain patients will be able to have treatment for just £2500 and that includes the drugs, so for some that dream of having a family may become just a little bit more affordable. Sky News came to the PET office to film our thoughts on this.

In other news, one woman's dream became her former partner's nightmare when he found out that she had forged his signature on a fertility clinic's consent form and become pregnant with his child. He lost his High Court case for damages for the cost of bringing his daughter up (as discussed elsewhere in BioNews this week). BBC Radio London interviewed me about this on Friday night.

This week our hopes for a dream line-up at our Annual Conference, 'Crossing Frontiers: Moving the Boundaries of Human Reproduction', were realised when Professor Magdalena Zernicka-Goetz - professor of mammalian development and stem cell biology at the University of Cambridge - confirmed that she was able to speak.

It isn't only the Prime Minister that has a bad day at the office – we had our own nightmare here with no phone line for two days, and no internet for more than half a day when fixing the phone somehow broke the internet! Of course this had to happen when we were trying to promote our next event, which is taking place in Sheffield.

There are many more dreams we would like to come true, such as holding more events outside London and filming them to make them more widely available, and we need your support to help us fulfil them.

We really treasure every donation we receive, and we do everything we can to maximise our resources to fulfil our vision of improving choices for people affected by infertility and genetic conditions.

But even so we need your help. Please help us reach our £4000 target via PayPal (click here), by text (text 'PROG23 £10' or any other amount to 70070), or by post (as detailed here). Or even better, become a Friend of PET as detailed here and give us a regular donation.

Thank you.



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Father loses claim against clinic after ex forged IVF consent

09 October 2017

By Antony Blackburn-Starza

Page URL: Appeared in BioNews 921

A man has lost his High Court claim for damages against a London IVF clinic for failing to obtain his consent for the use of embryos created with his former partner.

The man, named as ARB in court documents, is the father of two children born following fertility treatment in 2008 and 2011. He alleged that his partner, R – with whom he successfully underwent IVF in 2008 – had forged his signature on the consent forms required for a second treatment, after their relationship had broken down.

The couple had stored embryos with IVF Hammersmith, which ARB claimed were later thawed and used without his valid consent, leading to the birth of their second child. His 'seven figures' claim for damages for the upkeep of the second child was brought in contract against the clinic, which in turn sought an indemnity in the tort of deceit from R in the event that ARB's claim succeeded.

In his judgment, Mr Justice Jay held that although the clinic had exercised reasonable care in obtaining ARB's written consent, it was nevertheless under a strict contractual obligation not to proceed with embryo thawing and implantation without valid consent from both parties.

However, despite finding that the man's signature had probably been forged and that the clinic was in breach of its strict obligations, the judge rejected ARB's claim for legal policy reasons. Following case law that prevents parents from receiving damages for the costs of raising a healthy child, even if negligence is proven, he said:

'Whatever my personal response to this extraordinary case, acting in obedience with clear authority and principle compels me to uphold the clinic's submission that legal policy precludes all of ARB's pleaded claims.'

Expressing some unease with the outcome, Justice Jay said: 'Although he has lost this case, my judgment must be seen as a complete personal and moral vindication for ARB. The same, of course, cannot be said for R.'

Speaking after the ruling, the father involved in the case said that it 'never been about money' but had been about 'justice'. Jude Fleming, the chief operating officer of IVF Hammersmith, said that the clinic had changed its consent practices since the incident so that it would not happen again.

Representing the clinic, James Lawford Davies of Hempsons solicitors said: 'Clinics cannot presume that all their patients might be dishonest, but the case highlights the importance of having systems in place to ensure that both partners continue to agree to pursue treatment together, right up until the moment of transfer.'


24 July 2017 - by Georgia Everett 
A father is suing a London fertility clinic for £1 million after his ex-partner secretly conceived his baby via IVF following their split...
27 March 2017 - by Jennifer Willows 
A woman in Singapore has been awarded damages after an IVF clinic fertilised her eggs with a stranger's sperm instead of her husband's...
08 February 2016 - by Antony Blackburn-Starza 
A woman is suing healthcare professionals in the USA for failing to detect that she was a carrier of cystic fibrosis...
11 January 2016 - by Dr Mary Yarwood 
A US woman acting as a surrogate has filed a lawsuit to prevent a claim in damages being brought by the commissioning father after he requested she undergo a selective reduction...
23 March 2015 - by Chee Hoe Low 
A lecturer from London has been awarded nearly £40,000 in damages after his businesswoman ex-wife deceived him for over six years into believing that he was the biological father of her IVF-born son...


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Ireland to offer state-funded IVF

09 October 2017

By Georgia Everett

Page URL: Appeared in BioNews 921

For the first time couples in Ireland will be eligible for financial aid for fertility treatments, after the Government signed off new proposals last week.

Health Minister Simon Harris presented a draft of the Assisted Human Reproduction (AHR) Bill to Cabinet, which included an examination of how other countries, particularly the United Kingdom, have funded IVF treatments in order to see which scheme would work best in Ireland.

Mr Harris explained that the 'new legislation is the first time that a comprehensive package of measures has been drafted for the area of AHR as a whole. It has been long called for and is a very important milestone'.

The legislation will allow couples access to funding regardless of household income. Fertility treatment can be financially restrictive for couples who require treatment, as each IVF cycle costs approximately €4500-6000. The plans will allow the Government to decide how many funded cycles each couple is permitted to receive. Although all will have access to funding, it is understood that the number of funded cycles is likely to be means-tested.

The law will also permit gametes and embryos to be used for posthumous fertility treatment by the patient's surviving partner, if the applicable consents are in place. 

The proposed Assisted Reproduction Regulatory Authority, an independent body, will be set up to oversee the clinics and regulate the conditions surrounding gamete and embryo donations. Regulations are expected to include prohibition of commercial surrogacy and payment for gamete donations, and monitoring research involving embryos and stem cells.

The authority will have responsibility to ensure 'the welfare and best interests of children born through AHR' are maintained as part of the ethical framework of the legislation.

The Bill will have to undergo pre-legislative examination by the Oireachtas Committee, subject to Government approval, for the financial aid to be in place for 2019. However, there are many questions regarding the Bill that must be addressed before it can become law, including the level of subsidy and whether preimplantation genetic diagnosis will be covered for those who require it.

The Irish Independent | 04 October 2017
The Times | 04 October 2017
Irish Examiner | 04 October 2017
The Irish Times | 03 October 2017


30 October 2017 - by Shaoni Bhattacharya 
Access to IVF in England has worsened considerably over the last five years, according to data obtained by Fertility Fairness...
23 October 2017 - by Dr Michelle Rodgers 
Equity and access are among the most urgent issues for medically assisted reproduction. According to Ireland's Health Research Board, across Europe six countries offer full public funding, and 19 countries offer partial public funding...

02 October 2017 - by Julianna Photopoulos 
A complaint about radio and internet advertisements for a major IVF clinic in Ireland has been upheld by the Advertising Standards Authority for Ireland...
11 September 2017 - by Dr Mary Yarwood 
Cambridgeshire and Peterborough Clinical Commissioning Group has removed funding for free IVF treatment, despite being the county where the procedure was first developed 40 years ago...
11 September 2017 - by Professor Alan Trounson and Dr Karin Hammarberg 
In Australia, almost four percent of children are born as a result of assisted reproductive technologies. The comparatively high rate of ART use is in part due to costs being covered by the taxpayer-funded health insurance scheme, Medicare...
14 August 2017 - by Dr Kimberley Bryon-Dodd 
Funding cuts by the UK's National Health Service has meant that 13 areas in England have restricted or halted IVF treatment since the start of 2017, according to Fertility Network UK...


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Commission demands reform on experimental cell therapies

09 October 2017

By Jennifer Willows

Page URL: Appeared in BioNews 921

The reputation of stem cell research is being undermined by unscrupulous clinics offering unproven therapies, says a report published last week.

The Lancet Commission on Stem Cells and Regenerative Medicine brought together a number of leading scientists to review progress and consider challenges facing the discipline.

'Regenerative medicine offers transformative potential for the future of patient care. But that potential could be jeopardised by low-quality research and a loss of public trust in stem cell science,' commented The Lancet's editor-in-chief Dr Richard Horton.

In a sense, the field has been a victim of its own hype. 'Excitement about possible treatments for incurable diseases like multiple sclerosis and Parkinson's disease, inflated by media reports, has led desperate patients and families to a proliferating number of poorly regulated clinics peddling untested and potentially ineffective therapies,' the report says.

Stem cell therapies have been shown to be extremely effective in a number of skin and blood disorders, including patients with severe combined immunodeficiency – the former 'bubble babies' born without functioning immune systems – and look promising for other blood disorders such as beta-thalassemia (see BioNews 920). However progress has been significantly slower in other tissues.

Professor Giulio Cosso of the University of Manchester, who lead the commission, explained that in other organs, 'fixing' the cells is not enough.  

'As soon as you move to diseases that affect the muscles, the brain and the heart, you can't remove the diseased tissue. You add your healthy or cured cell to 95 that have not been cured,' he said.

The gap between public expectations and the number of proven treatments has allowed a market to spring up offering unproven treatments, often at high prices.

A well-known example is Davide Vanonni, who despite holding no medical or scientific qualifications offered stem cell treatments for neurological conditions in Italy before being convicted of conspiracy and fraud in 2015 (See BioNews 878).

Last month the FDA announced that it would be taking a tougher stance against US clinics offering unproven stem cell treatments (see BioNews 916). Treatments using only the patient's own cells are not subject to the same level of regulation as drugs under US law if the cells are only 'minimally manipulated' – and similar loopholes exist in many jurisdictions.

But the report argues that 'new therapies expose patients to risks, some of which are difficult to predict even with inbuilt safeguards' and that use in humans needs proper governance.

The commission's recommendations broadly fall into calls for improvement in four areas: science, funding models, governance and public engagement.

The Guardian | 04 October 2017
Manchester University | 05 October 2017
The Lancet | 04 October 2017


20 November 2017 - by Martha Henriques 
The US Food and Drug Administration has announced a fast-track review process for gene therapies and other regenerative medicine treatments...
20 November 2017 - by Dr Patrick Foong 
Stem cell research holds tremendous promise for the treatment of a wide variety of illnesses and conditions, from spinal cord injury to autism. However, much more work is necessary to translate stem cell research into safe and effective therapies...

04 September 2017 - by Rikita Patel 
The US Food and Drug Administration intends to investigate the use of unproven stem cell therapies being offered in the country's clinics...
10 July 2017 - by Lea Goetz 
International experts are calling for global action on unproven and potentially dangerous stem cell therapies and their misleading marketing to the public...
02 May 2017 - by Cathal Farrell 
The ability of stem cells to divide into different mature cell types has ignited the field of regenerative medicine. Stem cells promise to repair and regenerate damaged or diseased tissues without the need for orthodox medical or surgical interventions. However, there is disparity between the expectations held by the general public and some medical professionals versus the reality of the emerging clinical evidence...
21 November 2016 - by Rachel Siden 
Italian prosecutors are investigating whether discredited stem-cell entrepreneur Davide Vannoni is continuing to offer his unproven therapies in eastern Europe...
21 September 2015 - by Paul Waldron 
The Food and Drug Administration must do more to control clinics offering unproven stem cell treatments in the USA, say scientists....


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Gene therapy halts fatal child brain disease

09 October 2017

By Charlotte Spicer

Page URL: Appeared in BioNews 921

A new gene therapy has halted the progression of a fatal degenerative brain disease in a small study of affected boys.

Until now, the only effective treatment available for patients with the rare disease cerebral adrenoleukodystropy (ALD) was stem cell transplantation from a compatible donor. Yet the new therapy uses the patient's own stem cells.

'The impact of this trial has been phenomenal,' said Dr Florian Eichler at Massachusetts General Hospital in Boston, who co-led the study. 'Boys without a donor match for stem-cell transplant were often passing away within a year or two of their diagnosis. Now, with early diagnosis and gene therapy, these boys are living longer and some are thriving enough to play sports and participate in other normal day-to-day activities.'

In the global, multi-centre study, researchers collected stem cells from the blood of 17 boys aged four to 14 with the severe cerebral form of ALD, also known as 'Lorenzo's oil' disease after its depiction in a 1992 film of this name.

These patients have a faulty gene on the X chromosome which causes a build-up of fatty acids that damage the protective myelin sheath around nerves, leading to motor problems and loss of the ability to walk and talk within a few years.

The scientists used an inactive form of the HIV virus to insert a correct version of the faulty gene into the stem cells, before injecting them back into the bloodstream of the patient.

Two years after receiving the treatment, 15 of the 17 patients were functioning normally and free of major disability.

'This appears to be a terrific new therapy for many kids who had little hope before,' said Dr David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Centre in Massachusetts, and senior study author. He added that the use of a patient's own, gene-modified stem cells minimises the risk of significant side effects.

The work is ongoing with eight more boys being enrolled onto the study, and long-term follow-up of the patients.

The trial opens new avenues for using gene therapy in brain diseases. Dr Theodore Friedmann at the University of California San Diego School of Medicine, who was not involved in the study, told The New York Times: 'Many think the central nervous system is intractable and unapproachable. This study proves them wrong.'

The results of the clinical trial, published in The New England Journal of Medicine, brings this treatment closer to the clinic - with the biotechnology company bluebird bio which sponsored the study in talks with US and European regulators.

Vector blog, Boston Children's Hospital | 04 October 2017
Dana-Farber/Boston Children's | 04 October 2017
HealthDay News | 05 October 2017
The New England Journal of Medicine | 04 October 2017
New York Times | 05 October 2017


11 December 2017 - by Ewa Zotow 
Scientists revealed an innovative method which can be used to transplant blood-forming stem cells directly into the brain...
20 November 2017 - by Jennifer Willows 
A new clinical trial in California marks the first time that genome editing has been used inside the body, rather than on cells such as blood or skin which can be extracted, edited outside the body, and then replaced...

25 September 2017 - by Sarah Gregory 
A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis...
12 June 2017 - by Dr Rachel Huddart 
Life-threatening allergies and asthma could one day be treated by a single injection, say researchers who have successfully treated mice using gene therapy...
25 April 2016 - by Antony Blackburn-Starza 
A UK court has struck out a claim brought against a hospital for failing to diagnose a genetic disorder when testing the claimants' second cousin three years previously...
08 November 2009 - by Dr Rebecca Robey 
Gene therapy has been used to treat two young boys with a devastating and fatal brain disease called adrenoleukodystrophy (ALD). Two years after treatment, both boys showed signs that the disease had stopped progressing and that there were no serious side effects from the gene therapy. These results, published in the journal Science, show huge promise, both for the future treatment of ALD and for the revival of investigations into the use of gene therapy to treat a wide variety ...


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Researchers crack how BRCA1 gene raises cancer risk

09 October 2017

By Dr Loredana Guglielmi

Page URL: Appeared in BioNews 921

A major breakthrough in understanding how mutations in the BRCA1 gene raise cancer risk has been made by researchers in the USA.

The BRCA1 gene was known to have a role in repairing DNA damage, which can be caused by exposure to radiation, chemicals and other environmental stressors. Now researchers have shown how it interacts with another gene to trigger DNA repair.

'There have been about 14,000 papers written about BRCA1, and you would think we already know everything about the gene, but we don't,' said Professor Patrick Sung at the Yale Cancer Centre in New Haven, Connecticut, and study leader. 'Defining the mechanism of the BRCA-dependent DNA repair pathway will help scientists design drugs to kill cancer cells more efficiently.'

If a woman has a BRCA1 mutation, by the time she is 70 years old her chance of developing breast cancer is 80 percent, compared with 12 percent if she does not. There is also a higher risk of developing ovarian, prostate and pancreatic cancers.

Despite its discovery almost 20 years ago, the BRCA1 DNA repair mechanism remained unexplained.

'The only way to find out is to purify the proteins, study its properties … and reconstitute the DNA-repair reaction,' said Professor Sung to HealthDay News.

The team showed that BRCA1 interacts with another gene called BARD1 to trigger a cascade of genetic events to repair DNA breaks. The research, published in Nature, describes how mutations occurring in the complex BRCA1-BARD1 and not just the BRCA1 gene affect the DNA repair function, and so raise cancer risk.

The researchers say their findings could be used to determine how harmful a patient's BRCA1 mutation is, which could assist in planning treatment.

'I should be able to tell you what [the mutation] means,' Professor Sung said to HealthDay News. 'We should be able to tell whether it affects the DNA-repair process or not.'

Researchers first believed that BRCA1 and BRCA2 mutations could account for up to eight percent of breast or ovarian cancers. However, this is likely to be higher as in many cancers there is no evidence of mutation, but expression of BRCA genes is still silenced.

'Understanding this mechanism will provide the predictive power for doctors trying to establish a patient's personal risk of developing cancer,' Professor Sung said.


13 November 2017 - by Dr Loredana Guglielmi 
Researchers have introduced new criteria to screen for BRCA gene mutations that could prevent more than 10,000 cases of cancer and save more than 2000 lives in the next decade...
06 November 2017 - by Isobel Steer 
Machine learning has been used to develop a promising screening test for ovarian cancer, a team of US-based researchers has said...
30 October 2017 - by Annabel Slater 
Two studies have discovered 72 new genetic variants associated with the risk of developing breast cancer...

26 June 2017 - by Lea Goetz 
A study of almost 10,000 women carrying BRCA mutations has revealed the best estimate yet of their risk in developing breast and ovarian cancer...
12 June 2017 - by Dr Loredana Guglielmi 
A drug treatment for ovarian cancer has shown success against inherited breast cancer...
20 March 2017 - by Dr Molly Godfrey 
A genetic study of breast cancer patients suggests that existing drugs for treating rare breast and ovarian cancers may help more patients than previously thought...
25 April 2016 - by Dr Katie Howe 
Women with a mutation in the breast-cancer susceptibility gene BRCA1 may have reduced numbers of eggs left in their ovaries, according to a study led by Australian scientists...


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Human sperm maturation study may hold insights

09 October 2017

By Shaoni Bhattacharya

Page URL: Appeared in BioNews 921

Researchers hope that a study detailing the four stages of human sperm stem cell development may shed light on infertility and certain cancers.

The study is the first to look at sperm stem cell maturation specifically in humans, says the study team.

'This information yields new insights into how sperm stem cells function and develop under normal circumstances,' said Dr Bradley Cairns, lead study author at the Huntsman Cancer Institute at the University of Utah in Salt Lake City. 'We have built a very important framework we can now use to help us understand what happens when things go wrong, resulting in issues like infertility and cancer in men.'

The team examined the gene expression profile of sperm stem cells during development. They identified four distinct phases: starting with a 'quiescent' state, to a 'proliferation' state when stem cells divide, to a 'differentiation' state when they mature to become sperm.

Most notably the study found distinct transitions in factors that influenced the different cellular states including cell cycle regulators, transcription factors, and signalling factors.

The researchers suggest their findings may also help in the understanding of certain cancers. For example, men with infertility are at higher risk of testicular and prostate cancers.

'Our study sheds new light on how genes normally function in sperm stem cells,' said Dr Cairns. 'The next steps will be to use this knowledge to better understand what changes happen when sperm stem cells don't develop normally and instead convert into cancer cells.'

The findings were published in Cell Stem Cell.


25 September 2017 - by Shaoni Bhattacharya 
Men with obesity are more likely to have a poorer quality and quantity of sperm than men of a healthier weight, suggests a new study...
18 September 2017 - by Dr Kimberley Bryon-Dodd 
A type of chemical found in various personal beauty products and plastics may affect sperm and lower reproductive success, according to a new study...
07 August 2017 - by Caroline Law 
While media reports regularly remind us of women's biological clocks and warn of the dangers of women leaving it 'too late' to have children, until recently little attention has been paid to the role of men in timing when to have children, and the effect of age on male fertility...
31 July 2017 - by Dr Katie Howe 
Sperm counts of men in developed nations have fallen by 52 percent in the last 40 years...


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'CRISPR-Gold' repairs muscular dystrophy gene in mouse model

09 October 2017

By Emma Laycock

Page URL: Appeared in BioNews 921

Scientists have repaired the faulty gene in a mouse model of muscular dystrophy by using gold nanoparticles to deliver the genome editing tool CRISPR/Cas9.

The new system, termed CRISPR-Gold, overcomes some issues associated with viral vectors, including 'off-target' effects - the introduction of unwanted mutations into the genome (see BioNews 903).

CRISPR-Gold faithfully repaired the faulty dystrophin gene, implicated in causing Duchenne muscular dystrophy (DMD), and was a significant improvement over previous work that shortened the gene to give a milder disease (see BioNews 834).

'CRISPR-Gold and, more broadly, CRISPR-nanoparticles open a new way for safer, accurately controlled delivery of gene-editing tools,' said co-lead author Dr Irina Conboy at University of California, Berkeley. 'Ultimately, these techniques could be developed into a new medicine for Duchenne muscular dystrophy and a number of other genetic diseases.'

In nearly a third of DMD patients, the muscle-wasting disease is caused by a single base mutation or small deletion in the dystrophin gene. Scientists gave mice with DMD a single injection of CRISPR-Gold into their muscle tissues.

The gold nanoparticles encapsulated all of the genome editing tools: the enzyme Cas9, a guide RNA and donor DNA - and delivered them directly to cells. The guide RNA directed Cas9 to the dystrophin gene, where it cut the DNA. The cells then used the donor DNA as a template to edit the mutant sequence back to normal.

After two weeks, CRISPR-Gold had restored 5.4 percent of the dystrophin gene with minimal off-target effects. The mice also showed better agility and strength compared with control mice. The findings were published in the journal Nature Biomedical Engineering.

Clinical trials are needed to test whether CRISPR-Gold is effective in humans. Study co-authors Dr Kunwoo Lee, Dr Hyo Min Park and Professor Niren Murthy, all at UC Berkeley, have formed a start-up company GenEdit, focused on translating the technology to humans.

This work 'is a big step forward for biomedical application of CRISPR', Professor Vincent Rotello at University of Massachusetts, who not involved in the study, told The Scientist. However, he added, 'one of the challenges with the [CRISPR-Gold] system is it doesn't have systemic delivery'.

CRISPR-Gold currently requires localised injection directly into the affected tissue, limiting its therapeutic reach. Scientists are working on a next generation of particles that can deliver the genome editing system into tissues via the blood stream – preferentially targeting adult stem cells.

'There's a lot of work to be done to move to the point where we'll actually be able to cure diseases,' Professor Rotello said, 'but I think this shows the way forward'.

The latest developments in genome editing will be discussed at the session 'What Next for Genome Editing? Politics and the Public', at the Progress Educational Trust's upcoming public conference 'Crossing Frontiers: Moving the Boundaries of Human Reproduction'.

The conference is taking place in London on Friday 8 December 2017. Full details - including sessions, speakers and how to book your place - can be found here.

Genetic Engineering & Biotechnology News | 04 October 2017
Berkeley News | 03 October 2017
Fierce Biotech | 05 October 2017
Nature Biomedical Engineering | 02 October 2017
The Scientist | 02 October 2017


11 December 2017 - by Isobel Steer 
Scientists in California have used a modified form of the CRISPR/Cas9 genome editing approach to epigenetically treat diabetes, kidney disease and muscular dystrophy in mice...
20 November 2017 - by Helen Robertson 
For the first time ever, researchers have been able to film, in real-time, the activity of the CRISPR technique on a strand of DNA...

31 July 2017 - by Jenny Sharpe 
A safe and effective gene therapy treating Duchenne muscular dystrophy in dogs has been demonstrated...
11 January 2016 - by Jenny Sharpe 
Scientists in the USA have shown that the genome-editing technique CRISPR can improve muscle function in a mouse model of Duchenne muscular dystrophy...
16 November 2015 - by Jenny Sharpe 
Researchers have discovered a gene linked to Duchenne muscular dystrophy (DMD) after a dog bred to model the disease was found to have a protective mutation against it...
07 October 2013 - by Robert Meadowcroft and Neil Bennett 
The families of children and young people with the severe genetic muscle-wasting condition Duchenne muscular dystrophy received a heavy blow last week...


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Gene therapy restores vision in blind mice

09 October 2017

By Dr Kimberley Bryon-Dodd

Page URL: Appeared in BioNews 921

Gene therapy has restored some vision in mice blinded by retinitis pigmentosa, an inherited degenerative eye disease.

Retinitis pigmentosa is the most common cause of blindness in young people, and no effective treatments currently exist for patients with little sight left.

'There are many blind patients in our clinics and the ability to give them some sight back with a relatively simple genetic procedure is very exciting,' said Dr Samantha de Silva at the University of Oxford, the lead author of the study.

Diseases like retinitis pigmentosa cause gradual loss of light-sensitive cells in the eye's retina, eventually leading to blindness. Rather than trying to restore these cells, researchers used a viral vector to introduce a gene for melanopsin, a light-sensitive protein naturally found in the eye, into the remaining retinal cells.

The cells then produced melanopsin and became able to sense and respond to light, and send visual signals to the brain. The changes lasted longer than a year, around the lifetime of the mice used in the study.

'Our next step will be to start a clinical trial to assess this in patients,' said Dr de Silva.

The authors are hopeful that this common form of blindness may be able to at least be partially restored in the future by using this gene therapy technique.

'The effect of retinitis pigmentosa on families with the disease is devastating and we have spent many years working out new ways to slow the loss of sight and to begin restoring it,' said Professor Robert MacLaren also at the University of Oxford, whose group led the research. 'This new approach is exciting because by using a human protein that is already present in the eye we reduce the chances of causing an immune response.'

Professor Alan Boyd, President of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians, who was not involved in the study, said that no satisfactory treatments currently exist for patients with end-stage retinal degeneration, who have little sight left. Although human clinical trials could be a minimum of two to three years away, he said: 'These results are very encouraging and could lead to a potential treatment for end-stage blindness in humans.'

The study was published in the journal PNAS.


16 October 2017 - by Rikita Patel 
The US Food Drug and Administration advisory committee has backed the use of gene therapy to treat a hereditary disease for the first time...

22 May 2017 - by Dr Kimberley Bryon-Dodd 
A small, preliminary clinical trial has demonstrated safe and well-tolerated gene therapy to treat wet age-related macular degeneration...
03 May 2016 - by Sarah Gregory 
A trial of gene therapy for choroideremia, a rare form of inherited blindness, has partially restored the vision of several patients...
01 February 2016 - by Kulraj Singh Bhangra 
Researchers have used the CRISPR/Cas9 genome-editing technique to correct a genetic mutation that causes blindness...
24 August 2015 - by Antony Blackburn-Starza 
An experimental form of gene therapy to treat blindness has been showed to restore some vision in mice....
15 December 2014 - by Dr Rosie Gilchrist 
Scientists have used gene therapy to restore some sight to animals with visual impairment similar to retinosa pigmentosa, a type of human blindness...


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Podcast Review: Naked Genetics - Exposing the Contents of Your Genes

09 October 2017

By Cara Foley

Page URL: Appeared in BioNews 921

Exposing the Contents of Your Genes

Naked Genetics, 14 September 2017

Presented by Dr Kat Arney

'Exposing the Contents of Your Genes', Naked Genetics, Thursday 14 September 2017

'Would you donate your genome?'

Straight to the point, the introduction immediately grabbed my attention. Genome donation wasn't something I had even considered before listening to this podcast, so I was very curious to learn more.

Professor Colin Smith at the University of Brighton, UK, was introduced as one of the brave souls who had donated his genome.  My (rather naïve) initial expectation was that the whole process would be pretty similar to blood donation. But then came the revelation that Professor Smith had actually donated his genome to be published freely. Approximately 6.4 billion base pairs, sequenced and published online. Available for all and sundry to see.

Cue alarm bells. In this social media-heavy world, I am reluctant to even share whether I've munched on some toast, let alone reveal the 'the whole recipe book' of me to the world.

'This is the kind of thing that most people might feel a little bit nervous about, ' said Dr Kat Arney, the presenter of this Naked Genetics podcast, 'Exposing the contents of your genes'. Quite an understatement. Less than a minute in, I was already adamant that I would not be donating my genome.

But being the reasonable individual that I am (cough), I decided to keep listening and see if the subsequent chat could convince me otherwise.

The plot further thickens as Professor Smith admits that he has included his genome report on his personal website and it lists 'all of the notable features of [his] genome and [his] health risks, and potential health benefits.' I was shaking my head, incredulous, by this point.

Professor Smith attempted to justify what I perceived to be pure recklessness by saying, '… if the data is freely available and not behind a pay wall or not kept anonymous, there's going to be a lot more benefit to society'. Ah, societal benefit. That old chestnut.

But before I could even mull over this potential upside, Professor Smith identifies something unsettling that I had foolishly overlooked: 'The notion that you can keep genomic data anonymous is really probably not true, even though people are told that their genomic data would be anonymous. '

And he's right. Your genome is identifiably you. You will never be anonymous if you donate your genome to be sequenced and recorded. It's definitely different to blood donation.

The conversation turned to the ways in which you can actually donate your genome, and this piqued my interest further. With the Personal Genome Project UK, you have to donate your genome under 'open consent'. Donors have to pass an exam to show that they understand the basic principles of genetics and the risks involved with genome donation. The company provides a rather sizeable document that details the notoriously tricky concepts of informed consent and genetics along with data handling (it's actually quite informative; I read the whole thing because I was so intrigued).

'…your genome on its own doesn't really mean much. It's only when you compare it with other genomes,' states Professor Smith. That is true, but my genome is my own and it's pretty important to me, so I do feel protective over it.

While this part of the podcast was really engaging, Professor Smith's zeal for genome donation rendered it slightly one-sided. There could have been a greater exploration of the wider risks and benefits to help people to come to an educated answer to the titular question.

After frantically Googling 'genome donation' for a while, I moved onto the next section of interest in this podcast, 'Ethics and Gene Editing'.

Professor Jackie Leach Scully of Newcastle University begins by defining human genome editing. I was torn about this introduction; it did mean that genome editing isn't misinterpreted but it made for relatively uninteresting listening.

The ethics of genome editing is an inherently broad subject that can sometimes use rather dull conventional examples, but I thought Professor Leach Scully spiced up the chat sufficiently with talk about 'Frankenstein's monsters and mutant societies'.

She proceeds to make an important point in that science-fiction scenarios can seem so unlikely and far-fetched that we may simply shrug them off. But 'it's unwise to say these things won't happen and we can't often anticipate what some kind of really transformative intervention might come along.' This resonated with me as I definitely enjoy seeing evil, genetically modified super-humans on the rampage in movies, but I am decidedly less overjoyed at the prospect of them running riot in the real world. It's convinced me to think much harder about ethics in different contexts, which is a nice take-away from this podcast.

Unfortunately, the conversation becomes a little dry after this point. Professor Leach Scully and Dr Arney examine the debate on embryo research that began with the Warnock report, but disappointingly offer no new insights into it.

The conversation is steered towards the creation of a 'homogenous perfect human'. Professor Leach Scully's initial response is somewhat confusing, but I felt that she does finally get to the crux of it with this: 'If we think about that idea about perfect beauty, about the perfect kind of person, the desirable kind of person, that's changed such a lot over history – even recorded history.' It is not a new perspective by any means, but it's a very reasonable point to emphasise.

'Ethics and Gene Editing', for the most part, provides a fair and comprehensive discussion. It's just unexciting. It failed to trigger a desire in me to research the topics further, unlike the section on genome donation.

But overall, these two sections of the Naked Genetics podcast provided a lot of food for thought for me; surely, that makes them worth a listen.



02 October 2017 - by Sandy Starr 
What do patients and laypeople think and know about genome editing and its implications? What are the best ways for experts and others to discuss genome editing in public, so as to improve public understanding and avoid confusion? The Progress Educational Trust has set out to answer these questions, with its 'Basic Understanding of Genome Editing' project....
22 May 2017 - by Dr Peter Mills 
The Nuffield Council on Bioethics is seeking views on the reproductive uses of genome editing in humans. Regular readers of BioNews will know that genome editing (including with the CRISPR-Cas9 technique) offers a way of making precisely targeted modifications to DNA in living cells, harnessing the cells’ inbuilt repair mechanisms to repair a deliberate, double-stranded DNA break in a way that may either disable or introduce a functional DNA sequence...
19 December 2016 - by Dr Cathy Herbrand 
Baroness Mary Warnock was the first speaker at this year's Progress Educational Trust Annual Conference. She is best known for The Warnock report, which was written more than 30 years ago and has shaped legislation in the UK and around the world...
14 March 2016 - by Isobel Steer 
Genetic-testing company Ambry Genetics has launched a huge database of cancer-patient genetics, freely available to the public...


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Published by the Progress Educational Trust


Public Conference
8 December 2017

Speakers include

Professor Azim Surani

Professor Magdalena Zernicka-Goetz

Professor Robin Lovell-Badge

Sally Cheshire

Professor Guido Pennings

Katherine Littler

Professor Allan Pacey

Dr Sue Avery

Professor Richard Anderson

Dr Elizabeth Garner

Dr Andy Greenfield

Dr Anna Smajdor

Dr Henry Malter

Vivienne Parry

Dr Helen O'Neill

Dr César Palacios-González

Philippa Taylor

Fiona Fox

Sarah Norcross

Sandy Starr


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