Every question you have ever had about gene replacement for Rett syndrome is asked and answered in this webinar from the Rett Syndrome Research Trust (RSRT). How are genes made in a lab? How do you get the new gene into the body? Is gene replacement really one and done? What have we learned from animal studies? What is the risk associated with gene replacement? Is a cure even possible? Our guest presenter, Dr. Stuart Cobb, first published on Rett as co-author with Adrian Bird on the 2007 reversal paper.

0:00 Welcome by RSRT CEO Monica Coenraads and introduction of Dr. Stuart Cobb
2:46 RSRT's strategic approach to finding a cure for Rett syndrome
4:09 RSRT's approach to gene replacement
7:06 What is gene replacement therapy?
12:02 How gene therapy is delivered to brain cells
31:47 Is gene therapy really a "one and done" treatment for Rett syndrome?
35:29 Immune response to gene replacement therapy
40:04 Gene replacement therapy challenge: limited space for gene transport
42:29 Gene replacement therapy challenge specific to Rett syndrome: getting the right amount of MECP2
45:49 What we've learned from animal studies: gene replacement therapy can improve Rett symptoms
50:43 The variables associated with gene replacement therapy and what they mean for treatment of humans
52:36 Snapshot of the gene therapy landscape to bring treatments to market
55:08 How to participate in a gene replacement clinical trial
55:49 Gene replacement academic programs
56:15 Other forms of gene therapy
57:30 Risks of gene therapy and safety for clinical trials
1:01:46 What we have learned from other disorders about how gene replacement therapy might treat Rett syndrome
1:09:27 Questions and answers

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The Rett Syndrome Research Trust is singularly focused on a cure for Rett syndrome. Achieving success takes a deep understanding of the relevant science, an environment built for scientific collaboration, and experience with drug and business development. Today we are building on incredible scientific and industry momentum by implementing six genetic-based strategies that tackle Rett from every angle. Every single company pursuing a cure for Rett is able to do so because they leveraged discoveries and resources that were generated with RSRT funding. RSRT is the home of the Rett cure community, and we are confident that together we will cure Rett syndrome.