Your questions on gene therapy answered by the experts

Published Date
08/02/2018
Author
Jenny Sharpe
Category
Research
AAV virus

Dr Linda Popplewell and Professor George Dickson from Royal Holloway University took to Facebook last week to answer your questions on gene therapy. You can read the full transcript of the Q&A here.

Prof Dickson and Dr Popplewell are currently involved in a collaborative gene therapy project called UNITE-DMD. This aims to assess the safety of a gene therapy for Duchenne muscular dystrophy in a phase 1/2 clinical trial. During the Q&A, they spoke about this in more detail:

This trial is testing a type of microdystrophin [gene therapy] that is different from the others in trial in the US. The work is a joint effort between MDUK and AFM in France, and the Genethon labs are the sponsoring agents. Trials will take place in London at GOSH and in Newcastle.

We were delighted to see so many of our supporters from around the world engaging in the Q&A. Thank you to everyone who joined in – we hope you enjoyed it!

If you have any further questions about gene therapy or would like to suggest a research topic for future Q&As, please get in touch with our Research team at research@musculardystrophyuk.org

Further information

Learn about gene therapy using AAVs

Read the latest gene therapy news

Take a look at our Clinical Trials Information Service

 

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