Revolutionary gene therapy brings hope to retinal disease treatments


New Orleans, La. — Non-arteritic anterior ischemic optic neuropathy (NAION) is a condition where blood flow to the optic nerve has been impacted. It is not known what causes the reduced blood flow, but NAION causes abrupt vision loss. Currently, there are no effective treatments for NAION. This week at the Association for Research in Vision and Ophthalmology’s (ARVO) 2023 Annual Meeting in New Orleans, La., one study revealed how gene therapy could be a promising treatment in restoring vision.

Previously, lead researcher Bruce Ksander, PhD, and his collaborators at Harvard Medical School and Life Biosciences showed that epigenetic reprogramming via adeno-associated virus type 2 (AAV2) induced genes Oct4, Sox2, Klf4 (collectively known as OSK) could reverse cell aging in retinal ganglion cells (RGCs) in mice, thus restoring visual function. The next step was to determine whether this could be done in non-human primates (NHP) since they have similar visual systems to humans. To evaluate this, the researchers treated the eyes of NAION-induced African green monkeys with AAV2-OSK and gave them oral doxycycline daily.

The team found that OSK gene therapy was effective in restoring key aspects of visual function in NHPs. Ksander shared, “There is currently no successful treatment for patients with...NAION. [Our] study presented at ARVO from Harvard Medical School and Life Biosciences is the first report using a cellular rejuvenation approach via epigenetic reprogramming to significantly restore visual function in non-human primates with NAION-induced vision loss, confirming efficacy of this approach to treat retinal ganglion cell dysfunction.” The results provide proof-of-concept for the therapy’s further potential in treating other retinal disorders.

  • Abstract title: Epigenetic reprogramming - A novel gene therapy that restores vision loss in a nonhuman primate model of NAION
  • Presentation start/end time: Sunday, April 23, 12:00 – 12:15pm CT
  • Location: R02-R03
  • Presentation number: 474


The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include approximately 10,000 eye and vision researchers from over 75 countries. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders. Learn more at

Media contact:
Jenniffer Scherhaufer