Global treatment approaches, also called gene-agnostic therapies, are those that address pathophysiologic responses common to retinal degenerations, i.e., photoreceptor and RPE metabolic stress, dysfunction and, ultimately, cell death. Global treatment strategies may be classified as those that aim to maintain or replace dysfunctional photoreceptors and/or RPE (e.g., neuroprotective agents and stem cell therapies) or those that circumvent the dysfunctional photoreceptors entirely (e.g., bioelectronic retinal implants and optogenetics). The research and development costs of each gene-specific therapy are high relative to the number of individuals who can benefit from it. Gene agnostic treatments have the potential to benefit a significantly larger number of patients, including those for whom the genetic cause of their disease cannot be identified.

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Global Treatment Approaches Part 1: Neuroprotection and Stem Cell Therapy

  • Gareth D. Mercer,
  • Brian G. Ballios,
  • Peter J. Kertes

摘要

Global treatment approaches, also called gene-agnostic therapies, are those that address pathophysiologic responses common to retinal degenerations, i.e., photoreceptor and RPE metabolic stress, dysfunction and, ultimately, cell death. Global treatment strategies may be classified as those that aim to maintain or replace dysfunctional photoreceptors and/or RPE (e.g., neuroprotective agents and stem cell therapies) or those that circumvent the dysfunctional photoreceptors entirely (e.g., bioelectronic retinal implants and optogenetics). The research and development costs of each gene-specific therapy are high relative to the number of individuals who can benefit from it. Gene agnostic treatments have the potential to benefit a significantly larger number of patients, including those for whom the genetic cause of their disease cannot be identified.