scAAV1.tMCK.NTF3

scAAV1.tMCK.NTF3 is a gene therapy that uses a self-complementary adeno-associated virus type 1 (scAAV1) vector carrying the human neurotrophin-3 (NTF3/NT-3) gene under the control of a muscle-specific tMCK promoter. It is designed to treat Charcot-Marie-Tooth disease type 1A (CMT1A), a hereditary peripheral neuropathy. The therapy works by delivering the NTF3 gene to muscle cells, which then produce neurotrophin-3, a protein that supports the growth and survival of nerve cells. Pre-clinical studies in the Trembler J mouse model of CMT1A showed that this therapy increased myelinated fiber densities and improved compound muscle action potential (CMAP) amplitudes.

Other names

Neurotrophin-3 gene therapyNeurotrophin3 gene therapyNeurotrophin 3 gene therapyNT-3 gene therapyNT3 gene therapyNT 3 gene therapy