Emerging therapies for inherited retinal degeneration.

Abstract:

:Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous group of disorders, affect the function of photoreceptor cells and are among the leading causes of blindness. Recent advances in molecular genetics and cell biology are elucidating the pathophysiological mechanisms underlying these disorders and are helping to identify new therapeutic approaches, such as gene therapy, stem cell therapy, and optogenetics. Several of these approaches have entered the clinical phase of development. Artificial replacement of dying photoreceptor cells using retinal prostheses has received regulatory approval. Precise retinal imaging and testing of visual function are facilitating more efficient clinical trial design. In individual patients, disease stage will determine whether the therapeutic strategy should comprise photoreceptor cell rescue to delay or arrest vision loss or retinal replacement for vision restoration.

journal_name

Sci Transl Med

authors

Scholl HP,Strauss RW,Singh MS,Dalkara D,Roska B,Picaud S,Sahel JA

doi

10.1126/scitranslmed.aaf2838

subject

Has Abstract

pub_date

2016-12-07 00:00:00

pages

368rv6

issue

368

eissn

1946-6234

issn

1946-6242

pii

8/368/368rv6

journal_volume

8

pub_type

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