French researchers have developed gene therapy to fight a rare disease that affects the eyes: Leber’s hereditary optic neuropathy.
Have you ever heard of Leber’s hereditary optic neuropathy? LHON is a rare inherited disease that affects approximately 1 in 55,000 people worldwide. It appears rather in men, between 27 and 34 years old.
Caused by genetic mutations (and, in particular, by mutations in mitochondrial DNA that affect the production of a specific protein – ND4), LONH is characterized by loss of central vision which can progress to blindness – progressive or brutal. (Source: National Union of Ophthalmologists of France SNOF).
Better visual acuity thanks to gene therapy
Currently, there is no treatment that can cure or even slow down this rare pathology. However, researchers from Inserm, Sorbonne University and CNRS at the Institut de la Vision in Paris have designed a gene therapy approach for patients with LONI. Promising, gene therapy is a therapeutic strategy which consists of introducing genetic material into cells to treat a disease.
Scientists “tested” their gene therapy treatment on 37 patients with LONH in a clinical trial called “REVERSE”. Result? After injection of the gene therapy vector into one eye, improvement in bilateral vision (in both eyes) was observed in ¾ of patients. In short: the treated patients saw an average of 15 letters more on the tables used by ophthalmologists to assess visual acuity …
“This discovery opens up new perspectives for the treatment of patients with Leber’s optic neuropathy, whereas until today there was none that was really effective. Our results demonstrate the effectiveness of this genetical therapy innovative in the eye, which offers a true therapeutic option against blindness for patients“say the authors of this study published in the journal Science Translational Medicine.
Source : Inserm press release
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