Gene Therapy: Vision Restored

A landmark for the most celebrated, and in some ways the most disappointing, late 20th century arena of medical research should not go without comment.

Last week, at the Children’s Hospital of Philadelphia, University of Pennsylvania researchers announced that they had used a genetically engineered virus to introduce a gene into the retinas of young adults with a form of congenital blindness that has no treatment, Leber congenital amaurosis (LCA).  Within a few weeks all three patients had significant improvement in the treated eye, with no apparent side effects.

The only other successful gene transfer therapy trial took place in France, where children without functioning immune systems (”bubble-boy disease”) did achieve results, but several were diagnosed with leukemia thereafter.  Following the success at CHOP, geneticists may now turn increasingly to single-gene disorders as the first therapeutic targets.

Katherine A. High, M.D, a Howard Hughes Medical Institute investigator and one of the CHOP study leaders, has been investigating LCA for years.  For a field that has been racked by disappointment and controversy (the death of a gene therapy subject in 1998 at Penn shocked the scientific community),  the good news from CHOP is both a source of encouragement and a reminder of the patience that is required to translate theoretical principles in genetics into practical treatments.

Tags: genetics

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