Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy
“This represents the proof of principle that ex vivo lentiviral gene therapy into stem cells can correct a lysosomal storage disorder and this has been further applied to mucopolysaccharidosis type one... and could be applicable to other lysosomal storage …