Leber Hereditary Optic Neuropathy Treatment

Rare diseases affect a larger population than cancer and AIDS combined, with over 7,000 distinct conditions impacting more than 300 million globally. Approximately 1 in 10 individuals are affected by a rare disease, and it often requires several years to receive an accurate diagnosis, averaging around 4.8 years. Ap...

There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical...

Leber Hereditary Optic Neuropathy (LHON) is a significant contributing factor behind the increasing blindness burden among young population. The birth LHON prevalence is approximately 1 in 50,000 people as estimated by the Rare Disease Database. Leber Hereditary Optic Neuropathy is a common inherited condition c...