Gene therapy of storage disorders by retroviral and lentiviral vectors.

[1]  E. Bongarzone,et al.  Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[2]  D. Dickson,et al.  Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy , 2005, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

[3]  G. Comi,et al.  Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism , 2005, Nature.

[4]  M. Poe,et al.  Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. , 2005, The New England journal of medicine.

[5]  C. Wessig,et al.  Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy. , 2005, Human molecular genetics.

[6]  R. Moats,et al.  Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[7]  E. Medico,et al.  Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. , 2005, Blood.

[8]  L. Naldini,et al.  Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. , 2005, Human gene therapy.

[9]  A. Fischer,et al.  Efficacy of gene therapy for SCID is being confirmed , 2004, The Lancet.

[10]  F. Gage,et al.  Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[11]  Yan Hu,et al.  Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[12]  C. Caillaud,et al.  Prevention of neuropathology in the mouse model of hurler syndrome , 2004, Annals of neurology.

[13]  J. Cooper,et al.  Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis , 2004, Neurobiology of Disease.

[14]  C. von Kalle,et al.  LMO2 and gene therapy for severe combined immunodeficiency. , 2004, The New England journal of medicine.

[15]  W. Low,et al.  Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[16]  M. Sands,et al.  Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[17]  A. Annoni,et al.  Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. , 2004, Blood.

[18]  M. Poe,et al.  Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. , 2004, The New England journal of medicine.

[19]  M. Haskins,et al.  Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. , 2004, Molecular genetics and metabolism.

[20]  W. Hop,et al.  Long-Term Intravenous Treatment of Pompe Disease With Recombinant Human -Glucosidase From Milk , 2004 .

[21]  L. Naldini,et al.  Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. , 2004, The Journal of clinical investigation.

[22]  Y. Imai,et al.  Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[23]  J. Lemontt,et al.  Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[24]  P. Chang,et al.  Delivery of recombinant gene product to canine brain with the use of microencapsulation. , 2003, The Journal of laboratory and clinical medicine.

[25]  A. Crang,et al.  The presence of astrocytes in areas of demyelination influences remyelination following transplantation of oligodendrocyte progenitors , 2003, Experimental Neurology.

[26]  Y. Eto,et al.  Brain transplantation of genetically engineered human neural stem cells globally corrects brain lesions in the mucopolysaccharidosis type VII mouse , 2003, Journal of neuroscience research.

[27]  S. Ryazantsev,et al.  Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. , 2003, Molecular genetics and metabolism.

[28]  Shawn M. Burgess,et al.  Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration , 2003, Science.

[29]  A. Ballabio,et al.  The Multiple Sulfatase Deficiency Gene Encodes an Essential and Limiting Factor for the Activity of Sulfatases , 2003, Cell.

[30]  Yuqin Wang,et al.  Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. , 2003, The Journal of clinical investigation.

[31]  Angelo L. Vescovi,et al.  Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis , 2003, Nature.

[32]  S. Ryazantsev,et al.  Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[33]  H. Blau,et al.  Contribution of transplanted bone marrow cells to Purkinje neurons in human adult brains , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[34]  C. Peters,et al.  Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines , 2003, Bone Marrow Transplantation.

[35]  B. Crain,et al.  Transplanted bone marrow generates new neurons in human brains , 2003, Proceedings of the National Academy of Sciences of the United States of America.

[36]  R. Coggeshall,et al.  Region-specific generation of cholinergic neurons from fetal human neural stem cells grafted in adult rat , 2002, Nature Neuroscience.

[37]  C. von Kalle,et al.  Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[38]  I. Weissman,et al.  Engraftment of sorted/expanded human central nervous system stem cells from fetal brain , 2002, Journal of neuroscience research.

[39]  G. Aguirre,et al.  Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[40]  Paul Shinn,et al.  HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots , 2002, Cell.

[41]  P. Morel,et al.  Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[42]  A. Fischer,et al.  Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study , 2002, The Lancet.

[43]  R. McKay,et al.  Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease , 2002, Nature.

[44]  A. Mortellaro,et al.  Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning , 2002, Science.

[45]  L. Notarangelo,et al.  Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement , 2002, Nature Medicine.

[46]  Richard J Smeyne,et al.  Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells. , 2002, Blood.

[47]  Zeger Debyser,et al.  Characterization of lentiviral vector-mediated gene transfer in adult mouse brain. , 2002, Human gene therapy.

[48]  Paul M. McCray,et al.  Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[49]  R. Herzog,et al.  Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. , 2002, Blood.

[50]  A. Björklund,et al.  Migration patterns and phenotypic differentiation of long-term expanded human neural progenitor cells after transplantation into the adult rat brain. , 2002, Brain research. Developmental brain research.

[51]  J. Mallet,et al.  Long-Term Expression of β-Glucuronidase by Genetically Modified Human Neural Progenitor Cells Grafted into the Mouse Central Nervous System , 2002, Molecular and Cellular Neuroscience.

[52]  D. German,et al.  Neurodegeneration in the Niemann–Pick C mouse: glial involvement , 2002, Neuroscience.

[53]  L. Naldini,et al.  Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. , 2002, Human gene therapy.

[54]  M. Frotscher,et al.  Targeting gene-modified hematopoietic cells to the central nervous system: Use of green fluorescent protein uncovers microglial engraftment , 2001, Nature Medicine.

[55]  R. Sidman,et al.  Segregation of Human Neural Stem Cells in the Developing Primate Forebrain , 2001, Science.

[56]  R. D'Hooge,et al.  Bone marrow stem cell gene therapy of arylsulfatase A-deficient mice, using an arylsulfatase A mutant that is hypersecreted from retrovirally transduced donor-type cells. , 2001, Human gene therapy.

[57]  I. Martins,et al.  In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[58]  C. Walsh,et al.  Induction of tolerance to human factor VIII in mice. , 2001, Blood.

[59]  F. Ono,et al.  Widespread Distribution of Adenovirus-Transduced Monkey Amniotic Epithelial Cells after Local Intracerebral Injection: Implication for Cell-Mediated Therapy for Lysosome Storage Disorders , 2001, Cell transplantation.

[60]  J. Barranger,et al.  Lessons learned from the development of enzyme therapy for Gaucher disease , 2001, Journal of Inherited Metabolic Disease.

[61]  D. Jolly,et al.  Factors affecting long-term expression of a secreted transgene product after intravenous administration of a retroviral vector. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[62]  A. Consiglio,et al.  In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice , 2001, Nature Medicine.

[63]  J. Belmont,et al.  Enzyme-replacement therapy in mucopolysaccharidosis I. , 2001, The New England journal of medicine.

[64]  P. Chang,et al.  Somatic Gene Therapy for a Neurodegenerative Disease Using Microencapsulated Recombinant Cells , 2000, Experimental Neurology.

[65]  R. Proia,et al.  Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[66]  M. Haskins,et al.  Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[67]  L. Naldini,et al.  Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy , 2000, The journal of gene medicine.

[68]  A. Martínez-Serrano,et al.  Genetically Perpetuated Human Neural Stem Cells Engraft and Differentiate into the Adult Mammalian Brain , 2000, Molecular and Cellular Neuroscience.

[69]  V. Gieselmann,et al.  Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector , 2000, Gene Therapy.

[70]  J. Dick,et al.  Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.

[71]  D. Trono,et al.  Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. , 2000, Human gene therapy.

[72]  J. Bloch,et al.  Lentiviral Gene Transfer to the Nonhuman Primate Brain , 1999, Experimental Neurology.

[73]  James M. Wilson,et al.  Sustained Production of β-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice , 1999, Experimental Neurology.

[74]  W. Krivit,et al.  Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome. , 1999, Current opinion in hematology.

[75]  Monte A. Gates,et al.  Site-Specific Migration and Neuronal Differentiation of Human Neural Progenitor Cells after Transplantation in the Adult Rat Brain , 1999, The Journal of Neuroscience.

[76]  B. Davidson,et al.  Systemic and Central Nervous System Correction of Lysosomal Storage in Mucopolysaccharidosis Type VII Mice , 1999, Journal of Virology.

[77]  B. Torbett,et al.  Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. , 1999, Science.

[78]  D. Trono,et al.  Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery , 1998, Journal of Virology.

[79]  C. Elliger,et al.  Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus , 1998, Gene Therapy.

[80]  R. Sidman,et al.  Engraftable human neural stem cells respond to development cues, replace neurons, and express foreign genes , 1998, Nature Biotechnology.

[81]  L Naldini,et al.  Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector , 1997, Journal of virology.

[82]  Luigi Naldini,et al.  Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo , 1997, Nature Biotechnology.

[83]  J. Abkowitz,et al.  Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model. , 1997, Blood.

[84]  J. Wolfe,et al.  Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase , 1997, Nature Medicine.

[85]  É. Mezey,et al.  Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice. , 1997, Proceedings of the National Academy of Sciences of the United States of America.

[86]  R. McKay,et al.  Stem Cells in the Central Nervous System , 1997, Science.

[87]  R. D'Hooge,et al.  Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy. , 1996, Proceedings of the National Academy of Sciences of the United States of America.

[88]  F. Gage,et al.  In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector , 1996, Science.

[89]  L. Lockman,et al.  Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases , 1995, Cell transplantation.

[90]  E. Snyder,et al.  Neural progenitor cell engraftment corrects lysosomal storage throughout the MRS VII mouse brain , 1995, Nature.

[91]  E. Unger,et al.  Male Donor‐derived Cells in the Brains of Female Sex‐mismatched Bone Marrow Transplant Recipients: A Y‐Chromosome Specific In situ Hybridization Study , 1993, Journal of neuropathology and experimental neurology.

[92]  D. Pardoll,et al.  HEMATOPOIETIC CELL TRANSPLANTATION IN THE TWITCHER MOUSE: THE EFFECTS OF PRETRANSPLANT CONDITIONING WITH GRADED DOSES OF BUSULFAN , 1993, Transplantation.

[93]  A. Yeager,et al.  Hematopoietic Cell Transplantation after Administration of High-Dose Busulfan in Murine Globoid Cell Leukodystrophy (the Twitcher Mouse) , 1991, Pediatric Research.

[94]  A. Tessitore,et al.  Scientific category: CHEMOKINES Chemokine-induced recruitment of genetically modified bone marrow cells into the CNS of GM1-gangliosidosis mice corrects neuronal pathology , 2013 .

[95]  T. Daly AAV-mediated gene transfer to the liver. , 2004, Methods in molecular biology.

[96]  B. Davidson,et al.  Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system , 2003, Gene Therapy.

[97]  D. Kayda,et al.  Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[98]  Luigi Naldini,et al.  Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics , 2001, Nature Medicine.

[99]  J. Dick,et al.  Distinct classes of human stem cells that differ in proliferative and self-renewal potential , 2001, Nature Immunology.

[100]  G. Abor,et al.  Safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry's disease , 2001 .

[101]  F. Gage,et al.  Isolation, characterization, and use of stem cells from the CNS. , 1995, Annual review of neuroscience.

[102]  E. Neufeld,et al.  Lysosomal storage diseases. , 1991, Annual review of biochemistry.