Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis

This publication has 33 references indexed in Scilit:

• Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial
  Human Gene Therapy, 2011
• Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins
  Molecular Therapy, 2011
• Expression ofNpc1 in Glial Cells Corrects Sterility inNpc1 -/- mice
  Journal of Applied Genetics, 2009
• Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance
  American Journal of Human Genetics, 2007
• Structural Alterations of Epididymal Epithelial Cells in Cathepsin A—Deficient Mice Affect the Blood‐Epididymal Barrier and Lead to Altered Sperm Motility
  Journal of Andrology, 2007
• Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
  Blood, 2006
• Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria
  Gene Therapy, 2005
• Targeting macrophages with baculovirus‐produced lysosomal enzymes: implications for enzyme replacement therapy of the glycoprotein storage disorder galactosialidosis
  The FASEB Journal, 2004
• A single protocol to detect transcripts of various types and expression levels in neural tissue and cultured cells: in situ hybridization using digoxigenin-labelled cRNA probes
  Histochemistry and Cell Biology, 1993
• Expression of cDNA encoding the human “protective protein≓ associated with lysosomal β-galactosidase and neuraminidase: Homology to yeast proteases
  Cell, 1988