Gene therapy for mucopolysaccharidosis

[1] Gene therapy restores vision in a canine model of childhood blindness [J]. NATURE GENETICS, 2001, 28 (01) : 92 - 95

[2] A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus [J]. MOLECULAR THERAPY, 2002, 5 (02) : 125 - 132

[3] CORRECTION OF MUCOPOLYSACCHARIDOSIS TYPE-I FIBROBLASTS BY RETROVIRAL-MEDIATED TRANSFER OF THE HUMAN ALPHA-L-IDURONIDASE GENE [J]. HUMAN GENE THERAPY, 1992, 3 (04) : 371 - 379

[4] Molecular basis of mucopolysaccharidosis type IIIB in emu (Dromaius novaehollandiae):: An avian model of Sanfilippo syndrome type B [J]. GENOMICS, 2001, 74 (03) : 299 - 305

[5] ASHTON LJ, 1992, AM J HUM GENET, V50, P787

[6] Neuroprotective gene therapy for Huntington's disease using a polymer encapsulated BHK cell line engineered to secrete human CNTF [J]. HUMAN GENE THERAPY, 2000, 11 (12) : 1723 - 1729

[7] REPLACEMENT THERAPY FOR INHERITED ENZYME DEFICIENCY - MACROPHAGE-TARGETED GLUCOCEREBROSIDASE FOR GAUCHERS-DISEASE [J]. NEW ENGLAND JOURNAL OF MEDICINE, 1991, 324 (21) : 1464 - 1470

[8] BEHAVIORAL CONSEQUENCES OF BONE-MARROW TRANSPLANTATION IN THE TREATMENT OF MURINE MUCOPOLYSACCHARIDOSIS TYPE-VII [J]. JOURNAL OF CLINICAL INVESTIGATION, 1994, 94 (03) : 1180 - 1186

[9] A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome) [J]. GLYCOBIOLOGY, 1999, 9 (12) : 1389 - 1396

[10] CORRECTION OF MURINE MUCOPOLYSACCHARIDOSIS TYPE-VII (MPS-VII) BY BONE-MARROW TRANSPLANTATION AND GENE-TRANSFER THERAPY [J]. HUMAN GENE THERAPY, 1991, 2 (02) : 113 - 113

[1] Gene therapy restores vision in a canine model of childhood blindness [J]. NATURE GENETICS, 2001, 28 (01) : 92 - 95

[2] A deviant immune response to viral proteins and transgene product is generated on subretinal administration of adenovirus and adeno-associated virus [J]. MOLECULAR THERAPY, 2002, 5 (02) : 125 - 132

[3] CORRECTION OF MUCOPOLYSACCHARIDOSIS TYPE-I FIBROBLASTS BY RETROVIRAL-MEDIATED TRANSFER OF THE HUMAN ALPHA-L-IDURONIDASE GENE [J]. HUMAN GENE THERAPY, 1992, 3 (04) : 371 - 379

[4] Molecular basis of mucopolysaccharidosis type IIIB in emu (Dromaius novaehollandiae):: An avian model of Sanfilippo syndrome type B [J]. GENOMICS, 2001, 74 (03) : 299 - 305

[5] ASHTON LJ, 1992, AM J HUM GENET, V50, P787

[6] Neuroprotective gene therapy for Huntington's disease using a polymer encapsulated BHK cell line engineered to secrete human CNTF [J]. HUMAN GENE THERAPY, 2000, 11 (12) : 1723 - 1729

[7] REPLACEMENT THERAPY FOR INHERITED ENZYME DEFICIENCY - MACROPHAGE-TARGETED GLUCOCEREBROSIDASE FOR GAUCHERS-DISEASE [J]. NEW ENGLAND JOURNAL OF MEDICINE, 1991, 324 (21) : 1464 - 1470

[8] BEHAVIORAL CONSEQUENCES OF BONE-MARROW TRANSPLANTATION IN THE TREATMENT OF MURINE MUCOPOLYSACCHARIDOSIS TYPE-VII [J]. JOURNAL OF CLINICAL INVESTIGATION, 1994, 94 (03) : 1180 - 1186

[9] A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome) [J]. GLYCOBIOLOGY, 1999, 9 (12) : 1389 - 1396

[10] CORRECTION OF MURINE MUCOPOLYSACCHARIDOSIS TYPE-VII (MPS-VII) BY BONE-MARROW TRANSPLANTATION AND GENE-TRANSFER THERAPY [J]. HUMAN GENE THERAPY, 1991, 2 (02) : 113 - 113