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Gene therapy treatment for blindness

19th February 2020

The first patients have received a revolutionary new gene therapy that can restore eyesight as part of the NHS Long Term Plan.

Babies born with an inherited retinal disorder, known as Leber’s Congenital Amaurosis (LCA), have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

The life-changing treatment for children and adults – voretigene neparvovec – is the first in a new generation of gene therapies that can be directly administered to patients, in this case through an injection. Many patients in the trials recovered their night time vision with this treatment.