Viral vectors for gene delivery to the inner ear.

Abstract:

:Gene therapy using virus vectors to treat hereditary diseases has made remarkable progress in the past decade. There are FDA-approved products for ex-vivo gene therapy for diseases such as immunodeficiencies (e.g., SCID), and in vivo gene therapy for a rare blindness and neuro-muscular disease. Gene therapy for hereditary hearing loss has picked up pace in the past five years due to progress in understanding disease gene function as well as the development of better technologies such as adeno-associated virus (AAV) vectors, to deliver nucleic acid to target cells in the inner ear. This review has two major goals. One is to review the state of the art for investigators already working in preclinical cochlear gene therapy. The other is to present the language of vectorology and important considerations for designing and using AAV vectors to inner ear neurobiologists who might use AAV vectors in the cochlea for either therapeutic or basic biological applications.

journal_name

Hear Res

journal_title

Hearing research

authors

Maguire CA,Corey DP

doi

10.1016/j.heares.2020.107927

subject

Has Abstract

pub_date

2020-09-01 00:00:00

pages

107927

eissn

0378-5955

issn

1878-5891

pii

S0378-5955(19)30567-2

journal_volume

394

pub_type

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