Table 6 Illustrative timeline estimates for ocular gene transfer clinical trials or clinical application

“Bennett, an associate professor of ophthalmology at the University of Pennsylvania…said she hopes initial experiments in people can begin within about two years.”

2003

Leber congenital amaurosis clinical trials began in 2007, with phase-I results published in 2008 (Bainbridge et al.,[6]; Hauswirth et al.,[7]; Maguire et al.,[8]).

Anonymous: Gene therapy restores dogs’ eyesight, may treat blind. Houston Chronicle; 2001, April 28.

“Within five years [gene transfer] could be ready for testing on people who suffer age-related macular degeneration.”

2013

Phase I clinical trials were initiated in 2010 (NCT01678872).

Highfield R: Hope of genetic cure for failing eyesight.

The Daily Telegraph; 2008, April 28.

“This [phase-I gene transfer clinical trial for Leber congenital amaurosis] really paves the way for developing a treatment for people who have so far had no prospect of a cure,” said Robin Ali, an ophthalmologist at UCL… “Within two to three years it might be approved for use in the clinic.”

2010-2011

Leber congenital amaurosis is currently recruiting for a phase-III clinical trial (NCT00999609).

Sample I: Maze walk marks ‘huge advance’ in gene therapy for blindness. The Guardian; 2008, April 28.

“[achromatopsia] treatment could be as little as four or five years away.”

2016-2017

To be determined.

Hilpen K: The boy who sees in black and white. The Daily Express; 2012, March 6.