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Emerging therapies for inherited retinal degeneration.
Journal article

Emerging therapies for inherited retinal degeneration.

  • Scholl HP Department of Ophthalmology, University of Basel, 4056 Basel, Switzerland. hendrik.scholl@usb.ch.
  • Strauss RW Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD 21287, USA.
  • Singh MS Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD 21287, USA.
  • Dalkara D INSERM, UMR S 968, 75012 Paris, France.
  • Roska B Department of Ophthalmology, University of Basel, 4056 Basel, Switzerland.
  • Picaud S INSERM, UMR S 968, 75012 Paris, France.
  • Sahel JA INSERM, UMR S 968, 75012 Paris, France.
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  • 2016-12-09
Published in:
  • Science translational medicine. - 2016
Animals
Clinical Trials as Topic
Genetic Predisposition to Disease
Genetic Therapy
Humans
Molecular Biology
Optogenetics
Photoreceptor Cells
Retina
Retinal Degeneration
Stem Cell Transplantation
Stem Cells
Vision, Ocular
Visual Prosthesis
English 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.
Language
  • English
Open access status
closed
Identifiers
Persistent URL
https://folia.unifr.ch/global/documents/17733
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