Rett Syndrome: A History of Research and Therapeutic Outlooks
Type
Blog Post
Published
Nov. 19, 2020, 2:15 p.m.
...Lox-Stop cassette, resulting in the neurological deficits observed in Rett Syndrome. Restoring the MECP2...it, regardless of age or symptom severity. This exciting finding injected hope that a similar reversal ...MECP2 mutant alleles, in particular, have shown exciting and promising results for repairing mutations ...wild-type MECP2 is made inside the cells, but this is exciting progress in tackling the root cause of Rett Syndrome...the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery. Molecular Therapy...