Leber Hereditary Optic Neuropathy Treatment

Oct 16, 2023

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...

Aug 19, 2022

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...

Aug 19, 2019

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...