Mitochondrial gene therapy: AAV2-mediated direct delivery of nucleic acids to the mitochondrial matrix as a treatment for Lebers Hereditary Optic Neuropathy

被引：0

|

作者：

Huston, M. W. ^{[1
]}

Mastroberardino, P. G. ^{[2
]}

Wagemaker, G. ^{[3
]}

de Coo, I. F. M. ^{[1
]}

机构：

[1] Erasmus Univ, Med Ctr, Dept Neurol, Rotterdam, Netherlands

[2] Erasmus Univ, Med Ctr, Dept Genet, Rotterdam, Netherlands

[3] Hacettepe Univ, Stem Cell Res Ctr, Ankara, Turkey

来源：

HUMAN GENE THERAPY | 2014年 / 25卷 / 11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：

P153

引用

收藏

页码：A105 / A105

页数：1

相关论文

共 32 条

[31] Gene Delivery of ATP6 by a Mitochondrial Targeting Sequence Modification of AAV9 Capsid VP2 Rescues Cells with Mutated T8993G MtDNA Responsible for Neuropathy Ataxia and Retinitis Pigmentosa and Expresses in the Mouse CNS
Guy, John
Yuan, Huijan
MOLECULAR THERAPY, 2013, 21 : S148 - S148
[32] Preliminary safety and tolerability results of intravitreal administration of GS010, a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing human wildtype mitochondrial NADH dehydrogenase 4 (ND4) gene in patients with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial DNA mutation
Sahel, Jose Alain
Uretsky, Scott
Combal, Jean Philppe
Galy, Anne
Thomasson, Nitza
Fitoussi, Serge
Corral-Debrinsky, Marisol
Honnet, Geraldine
Vignal, Catherine
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2015, 56 (07)

← 1 2 3 4 →