Year: 2022
Type: article
Abstract: (Molecular Therapy 28, 2139–2149; October 2020) In the originally published version of this article, it was stated that “The National Institute of Standards and Technology has set the limit for an acceptable CRISPR off-targeting rate in patients at 25%. [56–59]” This percentage is incorrect, so we have elected to redact it entirely from the review. The authors regret this error. Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis PigmentosaMeng et al.Molecular TherapyAugust 26, 2020In BriefThere have been recent advances in gene replacement therapies for autosomal recessive and X-linked inherited retinal disorders, but treating autosomal dominant diseases remains challenging. Tsang and colleagues highlight recent developments in gene therapy for rhodopsin-mediated autosomal dominant retinitis pigmentosa. The therapeutic strategies discussed are applicable to all autosomal dominant disorders. Full-Text PDF Open Archive
Source: Molecular Therapy
Authors: Da Meng, Sara D. Ragi, Stephen H. Tsang
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,900
OpenAlex ID: W4282978782