Allele-Specific Deafness Gene Disruption through Discrimination of a Single Base Change by S. aureus Cas9KKH Prevents Progressive Hearing Loss after AAV Mediated Gene Delivery

被引：0

|

作者：

Gyorgy, Bence ^{[1
]}

Pan, Bifeng ^{[2
,3
]}

Nist-Lund, Carl ^{[2
,3
]}

Kleinstiver, Benjamin P. ^{[4
]}

Zaborowski, Mikolaj ^{[5
]}

Joung, J. Keith ^{[4
]}

Corey, David P. ^{[1
]}

Holt, Jeffrey R. ^{[2
,3
]}

机构：

[1] Harvard Med Sch, Neurobiol, Boston, MA USA

[2] Boston Childrens Hosp, Otolaryngol, Boston, MA USA

[3] FM Kirby Ctr Neurobiol, Boston, MA USA

[4] Massachusetts Gen Hosp, Mol Pathol Unit, Boston, MA 02114 USA

[5] Massachusetts Gen Hosp, Neurol, Boston, MA 02114 USA

来源：

MOLECULAR THERAPY | 2018年 / 26卷 / 05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：

364

引用

收藏

页码：170 / 170

页数：1

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[1] Allele-specific gene disruption through discrimination of a single base change by S. aureus Cas9-KKH prevents progressive hearing loss after AAV-mediated gene delivery
Gyorgy, B.
Nist-Lund, C.
Pan, B.
Asai, Y.
Karavitaki, K. D.
Kleinstiver, B. P.
Garcia, S. P.
Zaborowski, M. P.
Solanes, P.
Spataro, S.
Schneider, B. L.
Joung, J. K.
Geleoc, G. S. G.
Holt, J. R.
Corey, D. P.
HUMAN GENE THERAPY, 2019, 30 (11) : A11 - A12
[2] Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss
Gyorgy, Bence
Nist-Lund, Carl
Pan, Bifeng
Asai, Yukako
Karavitaki, K. Domenica
Kleinstiver, Benjamin P.
Garcia, Sara P.
Zaborowski, Mikolaj P.
Solanes, Paola
Spataro, Sofia
Schneider, Bernard L.
Joung, J. Keith
Geleoc, Gwenaelle S. G.
Holt, Jeffrey R.
Corey, David P.
NATURE MEDICINE, 2019, 25 (07) : 1123 - +
[3] Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss
Bence György
Carl Nist-Lund
Bifeng Pan
Yukako Asai
K. Domenica Karavitaki
Benjamin P. Kleinstiver
Sara P. Garcia
Mikołaj P. Zaborowski
Paola Solanes
Sofia Spataro
Bernard L. Schneider
J. Keith Joung
Gwenaëlle S. G. Géléoc
Jeffrey R. Holt
David P. Corey
Nature Medicine, 2019, 25 : 1123 - 1130