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

[1] A Mutation-Independent CRISPR/Cas9-based 'Knockout and Replace' Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
Liu, Chi-Hsiu
Wolf, Pavlina
Dong, Ruhong
Huang, Yan
Tabbaa, Diana
Marco, Eugenio
Duke, Brian
Pinilla, Andrea
Pant, Asha
D'Souza, Racheal
Newmark, Judith
Giannoukos, Georgia
Zhang, Kate
Timmers, Adrian
Shearman, Mark
Allocca, Mariacarmela
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
[2] A Mutation-Independent CRISPR/Cas9-Based `Knockout and Replace' Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
Liu, Chi-Hsiu
Wolf, Pavlina
Dong, Ruhong
Huang, Yan
Tabbaa, Diana
Marco, Eugenio
Duke, Brian
Pinilla, Andrea
Pant, Asha
D'Souza, Racheal
Newmark, Judith
Giannoukos, Georgia
Zhang, Kate
Timmers, Adrian
Shearman, Mark S.
Allocca, Mariacarmela
MOLECULAR THERAPY, 2022, 30 (04) : 572 - 573
[3] Advances towards a Dual AAV CRISPR-Cas9-Based 'Knockout and Replace' Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
Reyon, Deepak
MOLECULAR THERAPY, 2021, 29 (04) : 279 - 280
[4] Advances towards a dual AAV CRISPR-Cas9-based 'Knockout and Replace' strategy to treat rhodopsin-associated autosomal dominant Retinitis Pigmentosa
Allocca, Mariacarmela
Reyon, Deepak
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)
[5] Compact CRISPR/Cas12-Based Mutation Independent Therapy Strategy for Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa via Single AAVs
Li, Luyao
Kong, Xiangfeng
Li, Jinhui
Yang, Guixiang
Yao, Yuqin
Liu, Weizhi
Cai, Lingqiong
Chen, Jianlong
Wang, Xing
Wang, Shaoran
Shi, Linyu
Yang, Hui
MOLECULAR THERAPY, 2023, 31 (04) : 354 - 355
[6] Mutation-Independent suppression of rhodopsin in Autosomal dominant Retinitis Pigmentosa by siRNA
Cronin, TC
O'Reilly, M
O'Neill, B
Kiang, AS
Palfi, A
Kenna, PF
Farrar, GJ
Humphries, P
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2004, 45 : U586 - U586
[7] A Novel Mutation-Independent CRISPR-Based Gene Therapy to Treat Autosomal Dominant Retinitis Pigmentosa
Casini, Antonio
Sarracino, Ambra
Pezze, Laura
Bosetti, Maddalena
Bello, Matteo
Bertolini, Sally
Badowska, Kalina
Ciciani, Matteo
Esposito, Federica
Auricchio, Alberto
Cereseto, Anna
MOLECULAR THERAPY, 2024, 32 (04) : 332 - 333
[8] CRISPR/Cas9-based genome editing approaches for RP1 associated autosomal dominant Retinitis Pigmentosa
Collin, Caitlin
Liu, Qin
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2021, 62 (08)
[9] Pharmacological strategies for treating misfolded rhodopsin-associated autosomal dominant retinitis pigmentosa
Xi, Yibo
Chen, Yuanyuan
NEURAL REGENERATION RESEARCH, 2022, 17 (01) : 110 - 112
[10] Pharmacological strategies for treating misfolded rhodopsin-associated autosomal dominant retinitis pigmentosa
Yibo Xi
Yuanyuan Chen
Neural Regeneration Research, 2022, 17 (01) : 110 - 112

← 1 2 3 4 5 →