Periodic Reporting for period 1 - GT-GM1 (Ex vivo gene therapy for GM1-gangliosidosis)

GM1 is a genetic disorder that occurs when a person has very low amounts of a vital enzyme, β-galactosidase. The absence of this enzyme affects nerve cells (called neurons) in the brain and spinal cord, components of the central nervous system (CNS). GM1 gangliosidosis...