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Restoring vision using optogenetics without being blind to the risks.

Alexander R Harris1, Frederic Gilbert2

  • 1Aikenhead Centre for Medical Discovery, ARC Centre of Excellence for Electromaterials Science, Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, Melbourne, VIC, 3010, Australia. alexrharris@gmail.com.

Graefe'S Archive for Clinical and Experimental Ophthalmology = Albrecht Von Graefes Archiv Fur Klinische Und Experimentelle Ophthalmologie
|November 1, 2021
PubMed
Summary
This summary is machine-generated.

Optogenetics offers a potential treatment for retinitis pigmentosa, an incurable eye disease. Early human trials using gene therapy and special goggles are underway, raising important ethical considerations for patient consent and fair treatment.

Keywords:
Clinical trialEthicsInformed consentOptogeneticsRetinitis pigmentosa

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Area of Science:

  • Ophthalmology
  • Neuroscience
  • Genetics

Background:

  • Retinitis pigmentosa is a degenerative retinal disease causing severe vision loss due to photoreceptor cell death.
  • Optogenetics presents a novel therapeutic strategy by introducing light sensitivity to retinal neurons.
  • First-in-human clinical trials for optogenetic therapy in retinitis pigmentosa have recently been initiated.

Purpose of the Study:

  • To evaluate the feasibility and ethical implications of optogenetic treatments for retinitis pigmentosa.
  • To highlight the innovative gene therapy approach and its reliance on specialized light-delivery devices.
  • To underscore the necessity of addressing ethical issues in early-stage clinical research.

Main Methods:

  • Gene therapy to introduce light-sensitive proteins into retinal cells.
  • Utilizing specially designed goggles for targeted light stimulation of the treated eye.
  • Conducting first-in-human clinical trials (NCT02556736, NCT03326336).

Main Results:

  • First-in-human studies have commenced, marking a significant step in optogenetic therapy for retinitis pigmentosa.
  • The treatment involves irreversible gene modification and requires external light stimulation.
  • These trials necessitate careful ethical oversight due to the innovative nature of the intervention.

Conclusions:

  • Optogenetic therapy shows promise for retinitis pigmentosa, but ethical considerations are paramount.
  • Ensuring informed consent and fair participant treatment is critical for these advanced clinical trials.
  • Further research and ethical deliberation are essential as optogenetics moves toward clinical application.