Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles-Reference-Cited by-同舟云学术

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Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles

Published:2020-06 Issue: Volume:17 Page:156-173
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Sanjurjo-Soriano Carla,Erkilic Nejla,Baux David,Mamaeva Daria,Hamel Christian P.,Meunier Isabelle,Roux Anne-Françoise,Kalatzis Vasiliki

Funder

Saudi Ophthalmological Society

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference89 articles.

1. The molecular basis of human retinal and vitreoretinal diseases;Berger;Prog. Retin. Eye Res.,2010

2. Novel mutations in the long isoform of the USH2A gene in patients with Usher syndrome type II or non-syndromic retinitis pigmentosa;McGee;J. Med. Genet.,2010

3. Frequency of Usher syndrome in two pediatric populations: Implications for genetic screening of deaf and hard of hearing children;Kimberling;Genet. Med.,2010

4. An update on the genetics of Usher syndrome;Millán;J. Ophthalmol.,2011

5. Genetics and pathological mechanisms of Usher syndrome;Yan;J. Hum. Genet.,2010

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1. Glycogen myophosphorylase loss causes increased dependence on glucose in iPSC-derived retinal pigment epithelium;Journal of Biological Chemistry;2024-08

2. Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress;Biomolecules;2024-07-25

3. New Perspectives in Stem Cell Transplantation and Associated Therapies to Treat Retinal Diseases: From Gene Editing to 3D Bioprinting;Stem Cell Reviews and Reports;2024-02-06

4. The Potential and Application of iPSCs in Gene and Cell Therapy for Retinopathies and Optic Neuropathies;Acta Naturae;2024-01-17

5. Recent Therapeutic Progress and Future Perspectives for the Treatment of Hearing Loss;Biomedicines;2023-12-18

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