rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application

被引：14

作者：

Blanc, Fabian ^{[1
,2
,3
]}

Mondain, Michel ^{[1
,2
,3
]}

Bemelmans, Alexis-Pierre ^{[4
,5
]}

Affortit, Corentin ^{[1
,2
]}

Puel, Jean-Luc ^{[1
,2
]}

Wang, Jing ^{[1
,2
]}

机构：

[1] Hop St Eloi, Inst Neurosci Montpellier, INSERM, UMR 1051, Batiment INM,80 Rue Augustin Fliche,BP 74103, F-34091 Montpellier, France

[2] Univ Montpellier, 163 Rue Auguste Broussonnet, F-34090 Montpellier, France

[3] CHRU Montpellier, F-34090 Montpellier, France

[4] CEA, Direct Rech Fondamentale, Inst Biol Francois Jacob, Mol Imaging Res Ctr, F-92265 Fontenay Aux Roses, France

[5] Univ Paris Saclay, CEA, CNRS, Neurodegenerat Dis Lab,UMR9199, F-92265 Fontenay Aux Roses, France

来源：

JOURNAL OF CLINICAL MEDICINE | 2020年 / 9卷 / 02期

关键词：

rAAV; cochlear gene therapy; clinical application; genetic deafness; routes of delivery; serotypes; targets gene addition; RNAi; gene editing; ADENOASSOCIATED VIRUS VECTOR; GROWTH-FACTOR RECEPTOR; INNER-EAR; MOUSE MODEL; VESTIBULAR FUNCTION; ENDOLYMPHATIC SAC; HEARING-LOSS; IMMUNE-RESPONSES; GUINEA-PIGS; CELL;

D O I：

10.3390/jcm9020589

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Over the last decade, pioneering molecular gene therapy for inner-ear disorders have achieved experimental hearing improvements after a single local or systemic injection of adeno-associated, virus-derived vectors (rAAV for recombinant AAV) encoding an extra copy of a normal gene, or ribozymes used to modify a genome. These results hold promise for treating congenital or later-onset hearing loss resulting from monogenic disorders with gene therapy approaches in patients. In this review, we summarize the current state of rAAV-mediated inner-ear gene therapies including the choice of vectors and delivery routes, and discuss the prospects and obstacles for the future development of efficient clinical rAAV-mediated cochlear gene medicine therapy.

引用

页数：21

共 125 条

[1] The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9 [J].