TY - JOUR
T1 - Emerging therapies for inherited retinal degeneration
AU - Scholl, Hendrik P.N.
AU - Strauss, Rupert W.
AU - Singh, Mandeep S.
AU - Dalkara, Deniz
AU - Roska, Botond
AU - Picaud, Serge
AU - Sahel, José Alain
N1 - Funding Information:
J.-A.S. is supported by grants from Labex LifeSenses (ANR-10-LABX-65), Banque publique d'investissement, the Foundation Fighting Blindness (FFB), ERC Synergy grant "HELMHOLTZ," Pixium Vision, GenSight Biologics, Sanofi-Fovea, and Gene Signal. H.P.N.S. was supported by the FFB Clinical Research Institute (CRI); a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC (W81-XWH-07-1-0720 and W81XWH-09-2-0189), the Shulsky Foundation, and the Ocular Albinism Research Fund (Clark Enterprises Inc.); and an unrestricted grant to the Wilmer Eye Institute from Research to Prevent Blindness, Baylor-Johns Hopkins Center for Mendelian Genetics (National Human Genome Research Institute/NIH). R.W.S. was supported by the Austrian Science Fund (FWF #J3383-B23) and the Foundation Fighting Blindness Clinical Research Institute.
Publisher Copyright:
© 2016 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science.
PY - 2016/12/7
Y1 - 2016/12/7
N2 - 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.
AB - 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.
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U2 - 10.1126/scitranslmed.aaf2838
DO - 10.1126/scitranslmed.aaf2838
M3 - Review article
C2 - 27928030
AN - SCOPUS:85003550920
SN - 1946-6234
VL - 8
JO - Science translational medicine
JF - Science translational medicine
IS - 368
M1 - 368rv6
ER -