Lenadogene nolparvovec, an adeno-associated virus (AAV)-based gene therapy, was developed to address LHON and has been ...

But he wasn’t about to let mitochondrial disease Leber’s hereditary optic neuropathy (LHON) stand in his way, and he made his first appearance as part of Italy’s Paralympic team in 2016 ...

The testing revealed that Christiane also carries the faulty gene. It means that both her children, Ramus, 19, and 14-year-old Katrine, are at risk of developing LHON. “Me and my family were ...

With no other effective treatment options, Neurophth is developing two gene therapy candidates for LHON. Qiutang Li’s team uses a recombinant adeno-associated virus (AAV) to deliver the correct ...

One of these mitochondria-associated disorders is Leber hereditary optic neuropathy (LHON), which leads to a ... to the likely involvement of other genes and environmental factors.

is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based ...