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Gene Editing and Fragile X Syndrome: The Status of Research and what the Future Might Hold скачать в хорошем качестве

Gene Editing and Fragile X Syndrome: The Status of Research and what the Future Might Hold 5 years ago

CRISPR

CAS9

Gene editing

gene therapy

research

evidence

ethics

Fragile X Syndrome

Developmental Disability

Fragile X

FXS

Open University

University of Birmingham

Medical school

medical ethics

clinical trial

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Gene Editing and Fragile X Syndrome: The Status of Research and what the Future Might Hold

‘Fixing’ genes using editing has been getting lots of press, for both good and bad reasons. But as a potential alternative to a drug-based route to reboot fragile X brain cells, just how feasible might editing the fragile X gene be? Dr Hirst's talk aims to explain what is meant by gene editing and what it might mean for fragile X. Along the way he takes a look at some of the hurdles that have still to be overcome but also look at where major progress in other genetic therapies has been made. Dr Mark Hirst completed his PhD in the laboratory of David Porteous at the MRC Human Genetics Unit in Edinburgh in 1988 and from there moved to Oxford to work in the laboratory of Kay Davies within the institute of Molecular Medicine at the John Radcliffe Hospital. During this period he spent time working as a visiting PI in the laboratory of Charles Laird (FHCRC, Seattle, USA) and as a visiting researcher in the laboratory of Alan Wolffe (NIH, Bethesda, USA). Dr Hirst moved to the Open University in 1999 and became a fellow of the Royal Society of Biology in 2017. This was a talk presented at The Fragile X Society's 2019 Conference at the University of Birmingham Medical School, on 7th September 2019. For more information about our charity's conferences and events, visit our website: www.fragilex.org.uk

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