Best corrected visual acuity was sustained or improved in patients with choroideremia who underwent gene therapy in a phase 2 study, and the researchers suggest that BCVA may be a viable primary outcome in advanced cases.
Byron L. Lam, MD, and colleagues at Bascom Palmer Eye Institute administered a high dose of AAV2-REP1 (Nightstar Therapeutics) as a subfoveal injection in six men with genetically confirmed advanced choroideremia. REP1, or Rab escort protein 1, is a CHM gene product that is absent in choroideremia.
Intraoperative microscope-integrated OCT was used to guide (Read more...)
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