[Current therapeutic approaches in inherited retinal degeneration: from genes to chip]

Klin Monbl Augenheilkd. 2014 Mar;231(3):222-31. doi: 10.1055/s-0033-1360259. Epub 2014 Mar 21.
[Article in German]

Abstract

Different strategies for the treatment of inherited photoreceptor degeneration are currently being investigated, with each of these approaches facing specific challenges. Gene therapy, for instance, may be feasible only for genetically well-defined pathologies. However, inherited retinal disorders are genetically highly heterogeneous and early onset disorders may restrict the therapeutic window. The majority of currently developed molecular approaches aim at the reconstitution of physiologically important functions in RPE and photoreceptor. Neuroprotection attempts to prolong cell survival and proper function via sustained delivery systems that fulfil a long-term dynamic reservoir function for therapeutic neuroprotective compounds. Cell-based approaches include replacement strategies such as cell transplantation, the implantation of prosthetic devices or optogenetics. They aim at replacing lost neurosensory functions of the retina. This short review aims at providing an insight into current therapeutic strategies and future treatment options for retinal disorders. Pharmacological and nutritional support strategies are only briefly discussed as we focus here on molecular and prosthetic therapeutic approaches.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Combined Modality Therapy / instrumentation
  • Combined Modality Therapy / methods
  • Electric Stimulation Therapy / methods*
  • Genetic Therapy / methods*
  • Humans
  • Molecular Targeted Therapy / methods*
  • Neuroprotective Agents / therapeutic use*
  • Prostheses and Implants
  • Retinal Degeneration / diagnosis
  • Retinal Degeneration / genetics*
  • Retinal Degeneration / therapy*
  • Stem Cell Transplantation / methods*

Substances

  • Neuroprotective Agents