Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease

Antzeko izenburuak

• Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1: bFGFR-1 mediates AAV2 transduction of Purkinje cells
  nork: Belur, Lalitha R., et al.
  Argitaratua: (2008)
• Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
  nork: Belur, Lalitha R., et al.
  Argitaratua: (2017)
• The Defect in the Hurler and Scheie Syndromes: Deficiency of α-L-Iduronidase
  nork: Bach, Gideon, et al.
  Argitaratua: (1972)
• Direct Gene Transfer to the CNS Prevents Emergence of Neurologic Disease in a Murine Model of Mucopolysaccharidosis Type I
  nork: Wolf, Daniel A., et al.
  Argitaratua: (2011)
• Identification of mutations in the alpha-L-iduronidase gene (IDUA) that cause Hurler and Scheie syndromes.
  nork: Scott, H S, et al.
  Argitaratua: (1993)