MFSD2A lentiviral vector

MFSD2A lentiviral vector is a gene therapy construct designed to deliver and express the Major Facilitator Superfamily Domain Containing 2A (MFSD2A) protein in target cells. MFSD2A is a specialized sodium-dependent transporter located at the blood-brain barrier (BBB) that is essential for the uptake of lysophosphatidylcholine (LPC)-bound omega-3 fatty acids, particularly docosahexaenoic acid (DHA), into the central nervous system. Mutations in the MFSD2A gene result in a rare autosomal recessive neurodevelopmental disorder characterized by primary microcephaly, severe intellectual disability, and spasticity. This lentiviral vector is primarily utilized as a research tool to investigate lipid transport mechanisms and blood-brain barrier integrity, but it also serves as a preclinical candidate for gene replacement therapy to restore DHA transport and support brain growth in affected individuals.

Other names

MFSD2A gene therapyMFSD-2A gene therapyMFSD 2A gene therapyLenti-MFSD2ALenti-MFSD-2ALenti-MFSD 2Alentiviral-MFSD2Alentiviral-MFSD-2Alentiviral-MFSD 2A