Advances towards a dual AAV CRISPR-Cas9-based 'Knockout and Replace' strategy to treat rhodopsin-associated autosomal dominant Retinitis Pigmentosa

被引：0

作者：

Allocca, Mariacarmela ^{[1
]}

Reyon, Deepak ^{[1
]}

机构：

[1] Editas Med, Cambridge, MA USA

来源：

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE | 2021年 / 62卷 / 08期

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D O I：

暂无

中图分类号：

R77 [眼科学];

学科分类号：

100212 ;

摘要：

引用

页数：2

共 12 条

[1] 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
[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
Allocca, Mariacarmela
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (07)
[3] A Mutation-Independent CRISPR/Cas9-based 'Knockout and Replace' Strategy to Treat Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa
Liu, C. H.
Wolf, P.
Dong, R.
Huang, Y.
Tabbaa, D.
Marco, E.
Duke, B.
Pinilla, A.
Pant, A.
D'Souza, R.
Newmark, J.
Giannoukos, G.
Zhang, K.
Timmers, A.
Shearman, M. S.
Allocca, M.
HUMAN GENE THERAPY, 2022, 33 (23-24) : A16 - A16
[4] 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
[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] CRISPR/SaCas9-based gene editing rescues photoreceptor degeneration throughout a rhodopsin-associated autosomal dominant retinitis pigmentosa mouse model
Du, Wei
Li, Jiarui
Tang, Xin
Yu, Wenzhen
Zhao, Mingwei
EXPERIMENTAL BIOLOGY AND MEDICINE, 2023, 248 (20) : 1818 - 1828
[7] 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
[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] Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa
Li, Pingjuan
Kleinstiver, Benjamin P.
Leon, Mihoko Y.
Prew, Michelle S.
Navarro-Gomez, Daniel
Greenwald, Scott H.
Piprce, Eric A.
Joung, J. Keith
Liu, Qin
CRISPR JOURNAL, 2018, 1 (01): : 55 - 64
[10] 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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