lentiviral gene therapy for HPRT1

This lentiviral gene therapy is an investigational treatment for Lesch-Nyhan Disease (LND), a rare X-linked disorder caused by deficiency of the enzyme hypoxanthine-guanine phosphoribosyltransferase (HPRT). Developed by researchers at UCLA, the therapy utilizes a self-inactivating lentiviral vector to deliver a codon-optimized HPRT1 cDNA sequence. A distinguishing feature of this construct is the addition of a signal peptide designed to facilitate the extracellular secretion of the HPRT enzyme. This approach aims to achieve cross-correction, where modified hematopoietic stem cells (HSCs) and their progeny (such as brain-resident macrophages) secrete the functional enzyme to be taken up by neighboring non-transduced cells, potentially ameliorating both the systemic metabolic complications and the severe neurological symptoms characteristic of the disease.

Other names

HPRT1 gene therapyHPRT-1 gene therapyHPRT 1 gene therapyLesch-Nyhan disease gene therapy