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Glossary

Vector

A delivery system used in the laboratory to carry foreign DNA into a cell. Modified viruses are commonly used as vectors in gene therapy.



Articles using this Glossary Item

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Gene therapy shows 'mind-blowing' success in treating haemophilia A

18 December 2017 - by Sam Sherratt

A new gene therapy for haemophilia A has exceeded expectations in a recent clinical trial... [Read More]

New CRISPR technique reverses diabetes and kidney failure in mice without cutting DNA

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... [Read More]

Sick boy gets healthy new skin with genetically-altered stem cells

13 November 2017 - by Paul Waldron

A boy with a rare skin disease has been successfully treated by replacing most of his skin with grafts of stem cells modified by gene therapy.

FDA advisors back gene therapy for rare inherited blindness

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... [Read More]

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

09 October 2017 - by Emma Laycock

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... [Read More]

Gene therapy restores vision in blind mice

09 October 2017 - by Dr Kimberley Bryon-Dodd

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

Gene therapy reverses multiple sclerosis in mice

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... [Read More]

Gene therapy skin grafts for obesity and diabetes

07 August 2017 - by Annabel Slater

A proof-of-concept study in mice has demonstrated how skin grafts could deliver gene therapy for obesity and diabetes... [Read More]

Gene therapy reverses muscular dystrophy in dogs in world first

31 July 2017 - by Jenny Sharpe

A safe and effective gene therapy treating Duchenne muscular dystrophy in dogs has been demonstrated... [Read More]

Blood stem cells produced in the lab for the first time

22 May 2017 - by Paul Waldron

In two different studies, US scientists have succeeded in creating the stem cells which produce blood... [Read More]

Cancer fusion genes targeted with CRISPR

08 May 2017 - by Sarah Pritchard

Aggressive human prostate and liver tumours have been shrunk in mice by targeting a ‘fused’ gene mutation using CRISPR/Cas9... [Read More]

CRISPR gene-editing eliminates HIV in mice

08 May 2017 - by Emma Laycock

US scientists have used CRISPR gene-editing to remove HIV DNA from the genomes of living animals, eliminating further infection.

Sickle cell reversed with gene therapy in teenager

06 March 2017 - by Ayala Ochert

A teenage boy in France appears to have been cured of sickle cell disease using a gene therapy. He has been free of all signs of the disease for 15 months... [Read More]

CRISPR partially restores sight in blind rats

21 November 2016 - by Paul Waldron

Scientists in the US have used a genome-editing technique to partially restore vision in blind rats... [Read More]

Gene therapy halts early Alzheimer's disease in mice

17 October 2016 - by Annabel Slater

A new gene therapy has slowed the progress of early Alzheimer's disease in mice... [Read More]

Gene therapy reverses inherited blindness

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... [Read More]

Scientists create monkey with 'autism gene'

01 February 2016 - by Dr Julia Hill

A Chinese laboratory has created genetically modified monkeys that show symptoms of autism... [Read More]

'Suicide gene therapy' for prostate cancer shows promise

21 December 2015 - by Lone Hørlyck

A treatment for prostate cancer, combining radiotherapy and a new type of gene therapy, is both safe and effective, a study has found... [Read More]

Biotech company Editas to begin human gene editing in 2017

16 November 2015 - by Ari Haque

A US biotechnology start-up co-founded by two pioneers of CRISPR technology intends to begin gene editing in humans as part of an experimental treatment to target a rare genetic eye disorder... [Read More]

Gene mutation may hold key to muscular dystrophy treatment

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... [Read More]

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