Rett Syndrome: A History of Research and Therapeutic Outlooks
Type
Blog Post
...Rett syndrome experience regression of previously acquired skills such as speech, coordination, purposeful...programs in biopharma. AveXis, co-founded by Kaspar, acquired by Novartis and rebranded to Novartis Gene Therapies...adeno-associated viral (AAV) vector. The editase repaired half of the mutant MECP2 RNA in each of three ...Samaco RC, Zoghbi HY (2011) Adult Neural Function Requires MeCP2. Science 333:186–186 . https://doi.org/10.1126...