Predictably, the big presidential symposium at ASGCT reserved a slot for Jean Bennett, who led one of the three teams that have tested a gene transfer strategy for a rare genetic form of blindness, Leber’s Congenital Amaurosis (LCA). Unpredictably, however, Bennett trotted out one of her “treated” patients, Cory Haas, along with his two parents,… Continue reading ASGCT, continued: Eyes on Stage
Tag: LCA
Ark, Troubled Waters, and Rainbows for Gene Transfer
This morning I awoke to a news report by National Public Radio’s Joe Palca on promising developments in gene transfer. In it, Palca provided a good account of the field’s travails, as well as some encouraging developments in the last few years. The story ended with the prediction that the coming “months and years” would… Continue reading Ark, Troubled Waters, and Rainbows for Gene Transfer
The Vision Thing: Update on LCA
Last year’s “big ticket” item at ASGT was results from the first three patients in two gene transfer trials testing nearly identical products against a rare form of congenital blindness, Leber’s Congenital Amaurosis (LCA). I previously blogged (here and here and here and elsewhere) on the controversial decision to move the intervention into children, given the novelty… Continue reading The Vision Thing: Update on LCA
ASGT in San Diego
This year’s annual meeting of the American Society of Gene Therapy is in San Diego. I’ve been to several interesting talks thus far, and plan to post entries on a few. For now, here’s an overview of some major (or some not so major) clinical developments in gene transfer that are being reported at this… Continue reading ASGT in San Diego
Kid Time for LCA gene transfer?
(…continued from previous posts). The promising results, and the surge of expectation they generated, will be used by the investigators and many advocates for the blind to defend pursuing the remainder of the study in children. After all, children stand a better chance of benefiting, because their retinas have not yet degenerated. Traditionally, bioethicists have… Continue reading Kid Time for LCA gene transfer?
Imprinting Expectation
(…continued from previous post) Whatever the ambiguity and severe limitations in terms of sample size and interpretability, the NEJM studies have unleashed a torrent of expectation. Accoding to the U.K. Guardian, “an 18-year-old man… has amazed doctors by navigating a maze…” and the technique “represented a ‘huge advance.’” The Washington Post’s Rick Weiss was harshly… Continue reading Imprinting Expectation
Seeing Light?
…continued from previous post: So did the researchers make the blind see? Any suggestion that vision has been restored is premature. The authors of both reports acknowledge that measures of vision are subjective and visual improvements might reflect a placebo effect. What can be said– at least of the U.S. study– is that the pupil… Continue reading Seeing Light?
Safety First: Two Gene Transfer Studies Against Blindness
…continued from previous post: The preclinical studies supporting these human trials are about as good as it ever gets in translational research: numerous large animals (dogs) were treated effectively, and their vision in treated eyes seems to have been restored out to as much as eight years. The NEJM papers report delivering nearly identical vectors… Continue reading Safety First: Two Gene Transfer Studies Against Blindness
Envisioning Gene Transfer: Report on Congenital Blindness Studies
This week, the New England Journal of Medicine published two preliminary trial reports using gene transfer against a rare form of congenital blindness– Leber’s Congenital Amaurosis type 2. Since Jean Bennett and coworkers at the University of Pennsylvania reported in 2001 successfully correcting visual deficiencies in dogs afflicted with a nearly identical genetic condition, the… Continue reading Envisioning Gene Transfer: Report on Congenital Blindness Studies