Community Update: Progress To A Cure | Rett Syndrome Research Trust |
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The Rett Syndrome Research Trust (RSRT) is pursuing, in parallel, six therapeutic strategies aimed at the underlying root cause of Rett syndrome. The RSRT research team provide an update on curative efforts underway. Please note that subtitles are available on this video in a variety of languages.
0:00 Welcome and introductions 2:06 Why a cure may be possible for Rett syndrome 7:49 RSRT's six strategies for a cure 9:49 Gene replacement strategy -- the strategy closest to the clinic 14:22 Gene editing strategy 17:14 MECP2 reactivation strategy 18:36 RNA editing strategy 19:34 RNA trans-splicing strategy 20:53 Protein replacement strategy 22:30 The MECP2 drug development pipeline -- where we are in the phases of development with the six strategies 24:38 RSRT's philosophy and approach to funding research and finding a cure 27:27 What will a cure look like for my child with Rett syndrome? 29:15 How RSRT's funding works and where RSRT's funding comes from 39:05 Questions and answers --------------------------------- 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. |