KOLOA, Hawaii —Voretigene neparvovec benefits patients with Leber congenital amaurosis and may help advance future gene therapies, according to a speaker at Retina 2020.
“The premise of gene therapy is to deliver a normal copy of the gene ... allowing that cell to produce the protein, which it could not produce at that point. What that means is that it is ideally performed prior to significant cell loss,” Aaron Nagiel, MD, PhD, said.
Gene therapy is very effective for autosomal and X-linked recessive conditions, he said.
“I think that we (Read more...)
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