Helios Gene Gun Particle Delivery for Therapy of Acid Maltase Deficiency

摘要: Autosomal recessive deficiency of lysosomal acid maltase (GAA) or glycogen storage disease type II (GSDII) results in a spectrum phenotypes including rapidly fatal infantile disorder (Pompe's), juvenile, and late-onset adult myopathy. The onset form presents as hypotonia with massive accumulation skeletal heart muscle, death due to cardiorespiratory failure. Adult patients the slowly progressive develop severe muscle weakness respiratory Particle bombardment is safe, efficient physical method which high-density, subcellular-sized particles are accelerated high velocity carry DNA into cells. Because it does not depend on specific ligand, receptor, biochemical features cell surfaces, particle-mediated gene transfer can be readily applied variety systems. We evaluated particle delivery system for therapy GSDII. utilized vector carrying CMV promoter linked human GAA cDNA. Hum...