Not exact matches
A new
gene therapy treatment has restored some sight in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because of a broken or missing
gene called RPE65,
retinal photoreceptor
cells malfunction
and eventually die.
In recent years, scientists have grown new
retinal cells from stem
cells and shown progress in developing an effective
gene therapy.
Dominic Eberle (Ader, TUD)-- «
Cell transplantation
and gene therapy approaches for the treatment of
retinal degenerative disorders» (2012)
Many of the most promising approaches fall into four categories: the
retinal prosthetic,
gene therapy, stem
cell treatments,
and a technique that uses optogenetics, a way to engineer nerves to fire in response to bursts of light.
Some researchers seek
gene therapies that might act more broadly on
retinal cells, perhaps by using
genes that produce proteins supporting the growth
and health of nerve
cells, which could be delivered before significant vision loss occurs.