https://www.selleckchem.com/products/OSI-906.html
Background Giant axonal neuropathy (GAN; ORPHA 643; OMIM# 25685 is a rare, hereditary, pediatric neurodegenerative disorder associated with intracellular accumulations of intermediate filaments (IFs). Validation of therapeutic efficacy and viral vector delivery systems with GAN knockout (KO) mouse models has provided the springboard for the development of a viral vector being delivered intrathecally in an ongoing Phase I gene therapy clinical trial for the treatment of children with GAN (https//clinicaltrials.gov/ct2/show/NCT02362438)
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