scAAV9-hIGF1

scAAV9-hIGF1 is a gene therapy product consisting of a self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding the human insulin-like growth factor 1 (hIGF1) gene. This therapy is designed for intramuscular or systemic delivery to achieve robust and long-term expression of IGF-1 in target tissues. The primary mechanism involves the transduction of cells with the hIGF1 gene, leading to increased local production of IGF-1 protein, which exerts neuroprotective and muscle-regenerative effects. Preclinical studies have demonstrated that scAAV9-hIGF1 can significantly reduce motor neuron loss, delay muscle atrophy, prolong survival in amyotrophic lateral sclerosis (ALS) mouse models, and improve muscle size and function in models of peripheral arterial disease. The therapeutic effect is partly mediated by upregulation of D-amino acid oxidase (DAO), which modulates D-serine levels and protects motor neurons from apoptosis[1][4][6].

Other names

scAAV9–IGF-1self-complementary adeno-associated viral vector 9 encoding human insulin-like growth factor 1