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 Medjournal_title
Science translational medicineauthors
Scholl HP,Strauss RW,Singh MS,Dalkara D,Roska B,Picaud S,Sahel JAdoi
10.1126/scitranslmed.aaf2838subject
Has Abstractpub_date
2016-12-07 00:00:00pages
368rv6issue
368eissn
1946-6234issn
1946-6242pii
8/368/368rv6journal_volume
8pub_type
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