Gene Therapy Treatment for Choroideremia

how gene therapy works

Researchers at the Nuffield Laboratory of Ophthalmology at the University of Oxford in the U.K. are treating Choroideremia (CHM) with gene therapy.

Choroideremia is a rare genetic disorder that affects the choroid and retina, causing degeneration and a loss of sight over the life of the individual. The rate of vision loss varies from person to person, but usually begins with night blindness as a child then the loss of acuity, depth perception, color perception and worsening of night blindness as they get older.

As we usually hear with these stories about genetic therapy, the goal of the therapy was simply to slow down or even stop this degeneration and gradual loss of vision, but researchers and patients have been happy to report that they’ve also seen some restoration of vision as well!

We’ve been following the gene therapy for Leber’s Congenital Amaurosis (LCA) for a while and this works the same way. The basic concept is to use a vector to insert healthy genes into the eye where the new genes can do the job the patient’s genes aren’t able to do themselves.

This treatment has been found to be both safe and effective and can be used to treat many genetic disorders that cause blindness, including age-related macular degeneration.

Read more at BBC news: Gene therapy ‘could be used to treat blindness’.

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