Inhibition of Postn Rescues Myogenesis Defects in Myotonic Dystrophy Type 1 Myoblast Model

Inhibition of Postn Rescues Myogenesis Defects in Myotonic Dystrophy Type 1 Myoblast Model

Myotonic dystrophy kind 1 (DM1) is an inherited neuromuscular illness brought on by expanded CTG repeats within the 3′ untranslated area (3’UTR) of the DMPK gene. The myogenesis course of is flawed in DM1, which is intently related to progressive muscle weak spot and losing. Regardless of many proposed explanations for the myogenesis defects in DM1, the […]

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Mucopolysaccharidosis kind I (MPS I) is an inherited metabolic dysfunction brought on by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The 2 present therapies [hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT)], are insufficiently efficient in addressing neurologic illness, partially as a result of lack of ability of lysosomal enzyme to cross the […]