HIV infection is associated with several forms of peripheral neuropathy, the most common being a distal symmetrical polyneuropathy due to HIV infection (‘DSP’). Direct viral effects are an important ...

Axonal loss, after it exceeds a certain threshold, can give rise to persistent neuropathy. There is also evidence that significant axonal loss can limit the therapeutic potential of any new drug.

1. In this dose-escalation study, using intrathecal gene transfer with scAAV9/JeT-GAN demonstrated potential improvement in motor function. 2. Adverse events related to gene therapy included scoliosis ...

Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of symptoms and a challenging prognosis. Now, a study published recently in the New ...

MONDAY, March 25, 2024 (HealthDay News) -- Intrathecal gene transfer with scAAV9/JeT-GAN may result in some benefit for children with giant axonal neuropathy, according to a study published in the ...

Giant axonal neuropathy is a rare, autosomal recessive, pediatric, polysymptomatic, neurodegenerative disorder caused by biallelic loss-of-function variants in GAN, the gene encoding gigaxonin.We c ...

Giant Axonal Neuropathy News and Research RSS. Investigational gene therapy shows promise for rare childhood neurodegenerative disease.

An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic ...

Conclusions: CAA mice display an enhanced inflammatory response within the endoneurium which may contribute to observed axonal degeneration, ultimately impairing sensorimotor function and gait.