CRISPRi-Mediated Treatment of Dominant Rhodopsin-Associated Retinitis Pigmentosa-Reference-Cited by-同舟云学术

CRISPRi-Mediated Treatment of Dominant Rhodopsin-Associated Retinitis Pigmentosa

Published:2023-12-01 Issue:6 Volume:6 Page:502-513
ISSN:2573-1599
Container-title:The CRISPR Journal
language:en
Short-container-title:The CRISPR Journal

Author:

Burnight Erin R.¹²,Wiley Luke A.¹²,Mullin Nathaniel K.¹²,Adur Malavika K.³,Lang Mallory J.¹²,Cranston Cathryn M.¹²,Jiao Chunhua¹²,Russell Stephen R.¹²,Sohn Elliot H.¹²,Han Ian C.¹²,Ross Jason W.³,Stone Edwin M.¹²,Mullins Robert F.¹²,Tucker Budd A.¹²^ORCID

Affiliation:

1. Institute for Vision Research, Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.

2. Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.

3. Department of Animal Science, Iowa State University, Ames, Iowa, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Biotechnology

Link

https://www.liebertpub.com/doi/pdf/10.1089/crispr.2023.0039

Reference54 articles.

1. Clinically Focused Molecular Investigation of 1000 Consecutive Families with Inherited Retinal Disease

2. The cellular fate of mutant rhodopsin: quality control, degradation and aggresome formation

3. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

4. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy

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