An experimental gene therapy technique has demonstrated great promise in reversing blindness.
In research involving mice, a team of American scientists found a newly developed genetic therapy that addresses a protein deficiency linked to blindness restored the animals’ vision for at least six months.
The findings, published in the journal Human Gene Therapy, pave the way for clinical trials of the technique in people with Leber congenital amaurosis-1 (LCA1) — a disorder that causes severe visual impairment beginning in infancy tied to a deficiency in the protein retGC1.
The researchers — Sanford Boye, Shannon Boye, from University of Florida College of Medicine, Gainesville, University of Oklahoma College of Medicine, Oklahoma City, and Salus University, Elkins Park, Penn — said the treatment targets the loss of cone function that occurs in the eyes of patients with LCA1.
"This study shows the tremendous potential of recombinant gene therapy for the effective treatment of genetic causes of vision loss," said Terence R. Flotte, M.D., the journal’s editor and dean of the University of Massachusetts Medical School, Worcester, Mass.
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