Rett Syndrome: A History of Research and Therapeutic Outlooks
Type
Blog Post
...medical journal. Decades later and after years of painstaking work trying to isolate the causative gene for...recently used the CRISPR/Cas9 system to both precisely insert the MECP2-R270X mutation into the MECP2 gene in...fine-tuning dosage so that enough wild-type MECP2 is made inside the cells, but this is exciting progress in tackling...:2<147::aid-ajmg6>3.0.co;2-o
Baker SA, Chen L, Wilkins AD, Yu P, Lichtarge O, Zoghbi HY (2013) An AT-Hook...