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

[31] Zebrafish as a Model to Evaluate a CRISPR/Cas9-Based Exon Excision Approach as a Future Treatment Option for EYS-Associated Retinitis Pigmentosa
Schellens, Renske
de Vrieze, Erik
Graave, Pam
Broekman, Sanne
Nagel-Wolfrum, Kerstin
Peters, Theo
Kremer, Hannie
Collin, Rob W. J.
van Wijk, Erwin
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, 22 (17)
[32] A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency
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Yang, Yang
Breton, Camilo
Bell, Peter
Li, Mingyao
Zhang, Jia
Che, Yan
Saveliev, Alexei
He, Zhenning
White, John
Latshaw, Caitlin
Xu, Chenyu
McMenamin, Deirdre
Yu, Hongwei
Morizono, Hiroki
Batshaw, Mark L.
Wilson, James M.
SCIENCE ADVANCES, 2020, 6 (07)
[33] Mutation-independent RNA replacement approach based on RHO-targeting ribozyme rescues rod photoreceptor function in mice model of autosomal dominant Retinitis pigmentosa (adRP)
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[34] Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa
Zhouhuan Xi
Abhishek Vats
José-Alain Sahel
Yuanyuan Chen
Leah C. Byrne
Nature Communications, 13
[35] Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa
Xi, Zhouhuan
Vats, Abhishek
Sahel, Jose-Alain
Chen, Yuanyuan
Byrne, Leah C.
NATURE COMMUNICATIONS, 2022, 13 (01)
[36] New mutation in the 3'-acceptor splice site of intron 4 in the rhodopsin gene associated with autosomal dominant retinitis pigmentosa in a Basque family
Reig, C
Alvarez, AI
Tejada, I
Molina, M
Arostegui, E
Martin, R
Antich, J
Carballo, M
HUMAN MUTATION, 1996, 8 (01) : 93 - 94
[37] Modeling Dominant and Recessive Forms of Retinitis Pigmentosa by Editing Three Rhodopsin-Encoding Genes in Xenopus Laevis Using Crispr/Cas9
Joanna M. Feehan
Colette N. Chiu
Paloma Stanar
Beatrice M. Tam
Sheikh N. Ahmed
Orson L. Moritz
Scientific Reports, 7
[38] Modeling Dominant and Recessive Forms of Retinitis Pigmentosa by Editing Three Rhodopsin-Encoding Genes in Xenopus Laevis Using Crispr/Cas9
Feehan, Joanna M.
Chiu, Colette N.
Stanar, Paloma
Tam, Beatrice M.
Ahmed, Sheikh N.
Moritz, Orson L.
SCIENTIFIC REPORTS, 2017, 7
[39] OCULAR FINDINGS ASSOCIATED WITH A RHODOPSIN GENE CODON-106 MUTATION - GLYCINE-TO-ARGININE CHANGE IN AUTOSOMAL DOMINANT RETINITIS-PIGMENTOSA
FISHMAN, GA
STONE, EM
GILBERT, LD
SHEFFIELD, VC
ARCHIVES OF OPHTHALMOLOGY, 1992, 110 (05) : 646 - 653
[40] Autosomal-dominant retinitis pigmentosa associated with an Arg-135-Trp point mutation of the rhodopsin gene - Clinical features and longitudinal observations
Pannarale, MR
Grammatico, B
Iannaccone, A
Forte, R
DeBernardo, C
Flagiello, L
Vingolo, EM
DelPorto, G
OPHTHALMOLOGY, 1996, 103 (09) : 1443 - 1452

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