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

[41] Dual AAV-Based 'Knock-Out-and-Replace' of RHO as a Therapeutic Approach to Treat RHO-Associated Autosomal Dominant Retinitis Pigmentosa (RHO adRP)
Diner, Benjamin A.
Dass, Abhishek
Nayak, Radhika
Flinkstrom, Zachary
Tallo, Thomas
DaSilva, Jen
Gotta, Gregory
Wang, Tongyao
Marco, Eugenio
Giannoukos, Georgia
Ramachandran, Shyam
De Erkenez, Andrea
Jin, Shengfang
Albright, Charles F.
Reyon, Deepak
MOLECULAR THERAPY, 2020, 28 (04) : 108 - 109
[42] CRISPR/Cas9-based Knockout Strategy Elucidates Components Essential for Type 1 Interferon Signaling in Human HeLa Cells
Urin, Victoria
Shemesh, Maya
Schreiber, Gideon
JOURNAL OF MOLECULAR BIOLOGY, 2019, 431 (17) : 3324 - 3338
[43] THR4LYS RHODOPSIN MUTATION IS ASSOCIATED WITH AUTOSOMAL-DOMINANT RETINITIS-PIGMENTOSA OF THE CONE-ROD TYPE IN A SMALL DUTCH FAMILY
VANDENBORN, LI
VANSCHOONEVELD, MJ
DEJONG, LAMS
RIEMSLAG, FCC
DEJONG, PTVM
GAL, A
BLEEKERWAGEMAKERS, EM
OPHTHALMIC GENETICS, 1994, 15 (02): : 51 - 60
[44] In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
Bakondi, Benjamin
Lv, Wenjian
Lui, Bin
Jones, Melissa K.
Tsai, Yuchun
Kim, Kevin J.
Levy, Rachelle
Akhtar, Aslam Abbasi
Breunig, Joshua J.
Svendseni, Clive N.
Wang, Shaomei
MOLECULAR THERAPY, 2016, 24 (03) : 556 - 563
[45] AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa
Shahin, Saba
Xu, Hui
Lu, Bin
Mercado, Augustus
Jones, Melissa K.
Bakondi, Benjamin
Wang, Shaomei
PHARMACEUTICS, 2022, 14 (04)
[46] CRISPR/Cas9 gene editing for retinitis pigmentosa (RP): use of a selective guide discriminating the mutant dominant P23H from the wild-type rhodopsin (Rho) allele.
Giannelli, G.
Luoni, M.
Massimino, L.
Broccoli, V.
HUMAN GENE THERAPY, 2016, 27 (11) : A119 - A119

← 1 2 3 4 5 →