Small Molecule-Based Inducible Gene Therapies for Retinal Degeneration

Shyamtanu Datta, Hui Peng, John D. Hulleman

Research output: Chapter in Book/Report/Conference proceedingChapter

5 Scopus citations


The eye is an excellent target organ for gene therapy. It is physically isolated, easily accessible, immune-privileged, and postmitotic. Furthermore, potential gene therapies introduced into the eye can be evaluated by noninvasive methods such as fundoscopy, electroretinography, and optical coherence tomography. In the last two decades, great advances have been made in understanding the molecular underpinnings of retinal degenerative diseases. Building upon the development of modern techniques for gene delivery, many gene-based therapies have been effectively used to treat loss-of-function retinal diseases in mice and men. Significant effort has been invested into making gene delivery vehicles more efficient, less toxic, and non-immunogenic. However, one challenge for the treatment of more complex gain-of-function diseases, many of which might be benefited by the regulation of cellular stress-responsive signaling pathways, is the ability to control the strategy in a physiological (conditional) manner. This review is focused on promising retinal gene therapy strategies that rely on small molecule-based conditional regulation and the inherent limitations and challenges of these strategies that need to be addressed prior to their extensive use.

Original languageEnglish (US)
Title of host publicationAdvances in Experimental Medicine and Biology
Number of pages5
StatePublished - 2019

Publication series

NameAdvances in Experimental Medicine and Biology
ISSN (Print)0065-2598
ISSN (Electronic)2214-8019


  • Chemical biology
  • Dihydrofolate reductase
  • Gene therapy
  • Retinal degeneration
  • Small molecule-based conditional regulation
  • Therapeutics
  • Trimethoprim

ASJC Scopus subject areas

  • General Biochemistry, Genetics and Molecular Biology


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