A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa

Loss-of-function mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). We previously presented skipping of USH2A exon 13 as a promising treatment paradigm for USH2A-associated RP. However, RP-associated mutations are often private, and evenly d...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Renske T.W. Schellens, Sanne Broekman, Theo Peters, Pam Graave, Lucija Malinar, Hanka Venselaar, Hannie Kremer, Erik De Vrieze, Erwin Van Wijk
Format:	Article
Language:	English
Published:	Elsevier 2023-06-01
Subjects:	MT: Oligonucleotides: Therapies and Applications USH2A antisense oligonucleotides CRISPR-Cas9 dual exon skipping photoreceptors
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123001373