This
article explains new technologies for the treatment of Leber Congenital
Amaurosis (LCA), a group of degenerative diseases of the retina which are
typically inherited through the autosomal recessive pattern. LCA is the most common cause of congenital
blindness in children, but these new gene therapy treatments have proven
promising.
In
the early 1990’s, scientists found many mutations which cause blinding diseases
including one in RPE65 which causes Type 2 Leber Congenital Amaurosis
(LCA2). Researchers at the University of
Pennsylvania also found it possible to transfer healthy genes into retinal
cells using viruses. Because retinal
cells are terminally differentiated at birth, this treatment method is especially
convenient and in the retina.
While
the results of the treatment are most dramatic in children, the visual function
of all treated patients has improved.
All patients have in fact requested bilateral treatment following
treatment in one eye. Ideally, treatment
should be delivered between 3 months and 6 years of age. The treatment has also been used to give
color-blind squirrel monkeys color vision.
The frontier for retinal gene therapy therefore is multifaceted and promising
in many ways.
Sources
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