AAV-NLS-PGC1α4

AAV-NLS-PGC1α4 is an **investigational gene therapy** that delivers a nuclear localization signal (NLS)-tagged **PGC1α4** isoform to skeletal muscle via an **adeno-associated virus (AAV) vector**. The designed gene construct increases nuclear retention and activity of PGC1α4, a short isoform of the transcriptional coactivator PGC1α that is strongly induced by resistance exercise and promotes muscle hypertrophy. In preclinical models, muscle-specific overexpression via AAV-NLS-PGC1α4 led to increased muscle fiber size, improved muscle strength, enhanced myoblast viability, proliferation, and differentiation, and alleviated aging-associated sarcopenia and metabolic dysfunction (such as insulin resistance, fatty liver, and increased adiposity). Mechanistically, this therapy improves muscle and systemic metabolism partly by inducing myokines IGF1 and METRNL and improves insulin signaling and glucose uptake in skeletal muscle[1][3][4][5].

Other names

nuclear localized PGC1α4 AAV gene therapyNLS-PGC1α4 AAVAAV-mediated NLS-PGC1α4