Emerging therapies for inherited retinal degeneration

Hendrik P.N. Scholl, Rupert W. Strauss, Mandeep S. Singh, Deniz Dalkara, Botond Roska, Serge Picaud, José Alain Sahel

Research output: Contribution to journalReview articlepeer-review

107 Scopus citations


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.

Original languageEnglish (US)
Article number368rv6
JournalScience translational medicine
Issue number368
StatePublished - Dec 7 2016

ASJC Scopus subject areas

  • Medicine(all)


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