Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...many distinct CRISPR therapies to match these different mutations. For each therapy, effectiveness and ...’s size makes it difficult to deliver via gene therapy, so researchers have set their sights on another...work with dystrophin exon skipping? For such a therapy to be successful, multiple requirements must be...minimal risk of off-target editing. In order for the therapy to persist over time, stem cell editing would be...in DMD, and it’s estimated that exon skipping therapies would be applicable to 80% of DMD patients.
...characterizes additional, unique facets of CRISPR DMD therapy. Long et al. show that AAV-CRISPR does not cause...represent a great step forward for CRISPR gene therapy, it’s important to realize that DMD is a simpler...