Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa-Reference-Cited by-同舟云学术

Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

Published:2011-04 Issue:4 Volume:19 Page:642-649
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Millington-Ward Sophia,Chadderton Naomi,O'Reilly Mary,Palfi Arpad,Goldmann Tobias,Kilty Claire,Humphries Marian,Wolfrum Uwe,Bennett Jean,Humphries Peter,Kenna Paul F,Farrar G Jane

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference36 articles.

1. Gene therapeutic approaches for dominant retinopathies;Farrar;Curr Gene Ther,2010

2. Effect of gene therapy on visual function in Leber's congenital amaurosis;Bainbridge;N Engl J Med,2008

3. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial;Hauswirth;Hum Gene Ther,2008

4. Safety and efficacy of gene transfer for Leber's congenital amaurosis;Maguire;N Engl J Med,2008

5. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial;Maguire;Lancet,2009

Cited by 129 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

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

2. Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency;Journal of Inherited Metabolic Disease;2024-04-06

3. Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa;Human Gene Therapy;2024-03-01

4. CRISPR/SaCas9-based gene editing rescues photoreceptor degeneration throughout a rhodopsin-associated autosomal dominant retinitis pigmentosa mouse model;Experimental Biology and Medicine;2023-10

5. Binocular benefit following monocular subretinal AAV injection in a mouse model of autosomal dominant retinitis pigmentosa (adRP);Vision Research;2023-05