Subscribe to the BioNews newsletter for free

Login
Advanced Search

Search for
BioNews

Like the Progress Educational Trust on Facebook




 

Gene therapy directed straight to the brain works in mice

11 December 2017

By Ewa Zotow

Appeared in BioNews 930

Scientists revealed an innovative method which can be used to transplant blood-forming (hematopoietic) stem cells directly into the brain. The technique may provide a quicker and more effective way of treating diseases of the central nervous system compared with the standard, indirect mode of delivery.

The technique, reported in Science Advances, involved transplanting donor-derived hematopoietic stem cells (HSCs) directly into the lateral ventricles of the brain, from where they could deliver therapeutic molecules to affected tissues.

Researchers in the USA and Italy used a mouse model of a lysosomal storage disease, a group of metabolic disorders affecting the central nervous system. The disease causes a build-up of material which leads to neurodegeneration.

Until now, the HSCs were administered intravenously and affected the brain indirectly through bone marrow engraftment, a process where stem cells make their way through the bloodstream to the bone marrow and start producing new blood cells. This process is slow and can be inefficient in treating disorders where fast action is required.

'The main issue with the conventional HSC transplant strategy has been the length of time needed for the therapy to take effect in the brain,' said study lead Dr Alessandra Biffi, at the Dana-Farber Cancer Institute, Boston Children's Hospital in Boston, Massachusetts. 'It can take up to a year for the genetically-engineered cell lineage to proliferate, spread and produce therapeutic effects in the brain—oftentimes, patients don't have the luxury of time to wait.'

The HSCs transplanted directly to the brain showed faster effects than the HSCs administered intravenously. The transplant led to more rapid generation of new myeloid cells, which help to decrease the build-up of the excess material responsible for the damage to the nervous system.

Importantly, the donor-derived cells showed the same characteristics as cells of healthy animals. The transplanted cells were also detected in the brain only and not in the other parts of the central nervous system.

The team hopes the work could pave the way for prevention and treatment of lysosomal storage diseases as well as other neurodegenerative disorders such as Alzheimer's or Parkinson's.

Although the method may seem too invasive to be used in humans '[the treatment] would be in line with currently-used clinical procedures that enable access to the brain for treatment' said Dr Biffi.

RELATED ARTICLES FROM THE BIONEWS ARCHIVE

09 October 2017 - by Charlotte Spicer 
A new gene therapy has halted the progression of a fatal degenerative brain disease in a small study of affected boys...
04 September 2017 - by Caroline Casey 
Neurons derived from human stem cells have successfully been used to treat and relieve symptoms of Parkinson's disease in a primate animal model...
06 February 2017 - by Caroline Casey 
A novel gene therapy technique has partially restored hearing and balance in deaf mice...
15 June 2015 - by Hannah Somers 
Two US studies have identified specific genetic mutations in gliomas which correlate with how the tumours will behave and respond to treatment...

HAVE YOUR SAY
Be the first to have your say.

You need to or  to add comments.

By posting a comment you agree to abide by the BioNews terms and conditions


- click here to enquire about using this story.

Published by the Progress Educational Trust
Advertise your products and services HERE - click for further details

Good Fundraising Code

Become a Friend of PET HERE and give the Progress Educational Trust a regular donation