Emerging therapies for inherited retinal degeneration

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

Research output: Contribution to journalReview article

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.

Original languageEnglish (US)
Article number368rv6
JournalScience Translational Medicine
Volume8
Issue number368
DOIs
StatePublished - Dec 7 2016

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Photoreceptor Cells
Retinal Degeneration
Molecular Biology
Visual Prosthesis
Optogenetics
Retinal Diseases
Blindness
Cell- and Tissue-Based Therapy
Genetic Therapy
Cell Biology
Stem Cells
Therapeutics
Clinical Trials

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Scholl, H. P. N., Strauss, R. W., Singh, M., Dalkara, D., Roska, B., Picaud, S., & Sahel, J. A. (2016). Emerging therapies for inherited retinal degeneration. Science Translational Medicine, 8(368), [368rv6]. https://doi.org/10.1126/scitranslmed.aaf2838

Emerging therapies for inherited retinal degeneration. / Scholl, Hendrik P N; Strauss, Rupert W.; Singh, Mandeep; Dalkara, Deniz; Roska, Botond; Picaud, Serge; Sahel, José Alain.

In: Science Translational Medicine, Vol. 8, No. 368, 368rv6, 07.12.2016.

Research output: Contribution to journalReview article

Scholl, HPN, Strauss, RW, Singh, M, Dalkara, D, Roska, B, Picaud, S & Sahel, JA 2016, 'Emerging therapies for inherited retinal degeneration', Science Translational Medicine, vol. 8, no. 368, 368rv6. https://doi.org/10.1126/scitranslmed.aaf2838
Scholl HPN, Strauss RW, Singh M, Dalkara D, Roska B, Picaud S et al. Emerging therapies for inherited retinal degeneration. Science Translational Medicine. 2016 Dec 7;8(368). 368rv6. https://doi.org/10.1126/scitranslmed.aaf2838
Scholl, Hendrik P N ; Strauss, Rupert W. ; Singh, Mandeep ; Dalkara, Deniz ; Roska, Botond ; Picaud, Serge ; Sahel, José Alain. / Emerging therapies for inherited retinal degeneration. In: Science Translational Medicine. 2016 ; Vol. 8, No. 368.
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