Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA

• Main Authors: Scott J. Dooley (Author), Devin S. McDougald (Author), Krishna J. Fisher (Author), Jeanette L. Bennicelli (Author), Lloyd G. Mitchell (Author), Jean Bennett (Author)
• Format: Book
• Published: Elsevier, 2018-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Ocular gene therapy with recombinant adeno-associated virus (AAV) has shown vector-mediated gene augmentation to be safe and efficacious in the retina in one set of diseases (retinitis pigmentosa and Leber congenital amaurosis (LCA) caused by RPE65 deficiency), with excellent safety profiles to date...