Investigational gene therapy shows promise for rare childhood neurodegenerative disease

Mar 20 2024NIH/National Institute of Neurological Disorders and Stroke An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy , was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health . Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age.

Carsten G. Bonnemann, M.D., senior author and chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section at the National Institute of Neurological Disorders and Stroke Mutations to the GAN gene result in an inability to break down intermediate filaments, which are cellular structures that make up the framework of nerve cell extensions called axons. Axons are essential for transmission of signals between brain cells. The disease name refers to the enlarged and bloated appearance of the axon under the microscope.

Health Health Latest News, Health Health Headlines

Similar News:You can also read news stories similar to this one that we have collected from other news sources.

SARS-CoV-2-associated ARDS can damage the heart without direct infectionSARS-CoV-2, the virus that causes COVID-19, can damage the heart even without directly infecting the heart tissue, a National Institutes of Health-supported study has found.
Source: NewsMedical - 🏆 19. / 71 Read more »

Inflammation-reducing drug shows no benefit for dry age-related macular degeneration in trialThe drug minocycline, an antibiotic that also decreases inflammation, failed to slow vision loss or expansion of geographic atrophy in people with dry age-related macular degeneration (AMD), according to a phase II clinical study at the National Eye Institute (NEI), part of the National Institutes of Health.
Source: medical_xpress - 🏆 101. / 51 Read more »