A Mutation-Independent CRISPR/Cas9-based 'Knockout and Replace' Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa

被引：0

作者：

Liu, C. H. ^{[1
]}

Wolf, P. ^{[1
]}

Dong, R. ^{[1
]}

Huang, Y. ^{[1
]}

Tabbaa, D. ^{[1
]}

Marco, E. ^{[1
]}

Duke, B. ^{[1
]}

Pinilla, A. ^{[1
]}

Pant, A. ^{[1
]}

D'Souza, R. ^{[1
]}

Newmark, J. ^{[1
]}

Giannoukos, G. ^{[1
]}

Zhang, K. ^{[1
]}

Timmers, A. ^{[1
]}

Shearman, M. S. ^{[1
]}

Allocca, M. ^{[1
]}

机构：

[1] Editas Med, Cambridge, MA USA

来源：

HUMAN GENE THERAPY | 2022年 / 33卷 / 23-24期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

OR43

引用

页码：A16 / A16

页数：1

共 46 条

[21] Mutation-independent gene knock-in therapy targeting 5′UTR for autosomal dominant retinitis pigmentosa
Hoang, Duc Anh
Liao, Baoshan
Zheng, Zongli
Xiong, Wenjun
SIGNAL TRANSDUCTION AND TARGETED THERAPY, 2023, 8 (01)
[22] Mutation-independent gene knock-in therapy targeting 5′UTR for autosomal dominant retinitis pigmentosa
Duc Anh Hoang
Baoshan Liao
Zongli Zheng
Wenjun Xiong
Signal Transduction and Targeted Therapy, 8
[23] Functional rescue in a mouse model for autosomal dominant Retinitis Pigmentosa following mutation-independent gene therapy
Millington-Ward, Sophia
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HUMAN GENE THERAPY, 2008, 19 (10) : 1128 - 1129
[24] Allele-Specific Knockout by CRISPR/Cas to Treat Autosomal Dominant Retinitis Pigmentosa Caused by the G56R Mutation in NR2E3
Diakatou, Michalitsa
Dubois, Gregor
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Meunier, Isabelle
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INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, 22 (05) : 1 - 21
[25] A mild phenotype of autosomal dominant retinitis pigmentosa is associated with the rhodopsin mutation Pro-267-Leu
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OPHTHALMIC GENETICS, 1997, 18 (02): : 63 - 70
[26] Ocular signs associated with a rhodopsin mutation (Cys-167→Arg) in a family with autosomal dominant retinitis pigmentosa
Simonelli, F
Rinaldi, M
Nesti, A
Testa, F
Rinaldi, E
Ciccodicola, A
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[27] A Mutation-Independent RNA Replacement Approach Based on a RHO-Targeting Trans-Splicing Ribozyme for the Treatment of Autosomal Dominant Retinitis Pigmentosa (adRP)
Kim, Jihyun
Kim, Ji-na
Cho, Minyoung
Jalligampala, Archana
McCall, Maureen A.
Lee, Seong-Wook
MOLECULAR THERAPY, 2023, 31 (04) : 183 - 183
[28] A NEW CODON 15 RHODOPSIN GENE MUTATION IN AUTOSOMAL-DOMINANT RETINITIS-PIGMENTOSA IS ASSOCIATED WITH SECTORIAL DISEASE
SULLIVAN, LJ
MAKRIS, GS
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ARCHIVES OF OPHTHALMOLOGY, 1993, 111 (11) : 1512 - 1517
[29] OCULAR FINDINGS ASSOCIATED WITH A RHODOPSIN GENE CODON-58 TRANSVERSION MUTATION IN AUTOSOMAL DOMINANT RETINITIS-PIGMENTOSA
FISHMAN, GA
STONE, EM
GILBERT, LD
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SHEFFIELD, VC
ARCHIVES OF OPHTHALMOLOGY, 1991, 109 (10) : 1387 - 1393
[30] CRISPR/Cas9-Mediated Allele-Specific Disruption of a Mutant Human Rhodopsin Gene in a Transgenic Mouse Line Modeling Autosomal Dominant Retinitis Pigmentosa
Li, Wenbo
Yu, Wenhan
Joe, Myung Kuk
Hiriyanna, Suja
Li, Tiansen
Wu, Zhijian
MOLECULAR THERAPY, 2019, 27 (04) : 118 - 118

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