Intraperitoneal inoculation of Sandhoff mouse neonates with an HIV-1 based lentiviral vector exacerbates the attendant neuroinflammation and disease phenotype

[1]  Raymond A. Dwek,et al.  Storage solutions: treating lysosomal disorders of the brain , 2005, Nature Reviews Neuroscience.

[2]  D. Kohn,et al.  HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys , 2005, Gene Therapy.

[3]  H. Federoff,et al.  beta-hexosaminidase lentiviral vectors: transfer into the CNS via systemic administration. , 2005, Brain research. Molecular brain research.

[4]  H. Federoff,et al.  Systemic FIV vector administration: transduction of CNS immune cells and Purkinje neurons. , 2003, Brain research. Molecular brain research.

[5]  H. Federoff,et al.  Transcriptional and posttranslational regulation of Cre recombinase by RU486 as the basis for an enhanced inducible expression system. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

[6]  B. Nuttin,et al.  Optimized lentiviral vector production and purification procedure prevents immune response after transduction of mouse brain , 2003, Gene Therapy.

[7]  I. Verma,et al.  Efficient production of human FVIII in hemophilic mice using lentiviral vectors. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.

[8]  A. Lever,et al.  Lentiviral vectors for gene delivery to normal and demyelinated white matter , 2003, Glia.

[9]  David A. Smith,et al.  Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis. , 2003, Brain : a journal of neurology.

[10]  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.

[11]  R. Proia,et al.  Molecular pathophysiology in Tay-Sachs and Sandhoff diseases as revealed by gene expression profiling. , 2002, Human molecular genetics.

[12]  W. Min,et al.  Endostatin gene transfer inhibits joint angiogenesis and pannus formation in inflammatory arthritis. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[13]  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.

[14]  M. Sands,et al.  Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer , 2001, Gene Therapy.

[15]  B. Rollins,et al.  Absence of Monocyte Chemoattractant Protein 1 in Mice Leads to Decreased Local Macrophage Recruitment and Antigen-Specific T Helper Cell Type 1 Immune Response in Experimental Autoimmune Encephalomyelitis , 2001, The Journal of experimental medicine.

[16]  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.

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

[18]  J. Crawley,et al.  A genetic model of substrate deprivation therapy for a glycosphingolipid storage disorder. , 1999, The Journal of clinical investigation.

[19]  B. Davidson,et al.  Gene therapy for lysosomal storage diseases. , 1998, Molecular therapy : the journal of the American Society of Gene Therapy.

[20]  J. Crawley,et al.  Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice. , 1998, The Journal of clinical investigation.

[21]  D. Peterson,et al.  Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors , 1997, Nature Genetics.

[22]  R. Proia,et al.  Mice deficient in all forms of lysosomal beta-hexosaminidase show mucopolysaccharidosis-like pathology. , 1997, Journal of neuropathology and experimental neurology.

[23]  Michael P. McDonald,et al.  Mice lacking both subunits of lysosomal β–hexosaminidase display gangliosidosis and mucopolysaccharidosis , 1996, Nature Genetics.

[24]  M. Perricaudet,et al.  Restoration of hexosaminidase A activity in human Tay-Sachs fibroblasts via adenoviral vector-mediated gene transfer. , 1996, Gene therapy.

[25]  J. Flax,et al.  Expression of human β–hexosaminidase α–subunit gene (the gene defect of Tay–Sachs disease) in mouse brains upon engraftment of transduced progenitor cells , 1996, Nature Medicine.

[26]  H. Munier-Lehmann,et al.  Function of the two mannose 6-phosphate receptors in lysosomal enzyme transport. , 1996, Biochemical Society transactions.

[27]  Michael P. McDonald,et al.  Mouse models of Tay–Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism , 1995, Nature Genetics.

[28]  F. E. Bertrand,et al.  Developmental Regulation of the Human Antibody Repertoire a , 1995, Annals of the New York Academy of Sciences.

[29]  W. Risau,et al.  Differentiation-dependent expression of proteins in brain endothelium during development of the blood-brain barrier. , 1986, Developmental biology.

[30]  G. Goldstein,et al.  In Vitro Studies of the Blood‐Brain Barrier Using Isolated Brain Capillaries and Cultured Endothelial Cells a , 1986, Annals of the New York Academy of Sciences.

[31]  R. Proia,et al.  Association of alpha- and beta-subunits during the biosynthesis of beta-hexosaminidase in cultured human fibroblasts. , 1984, The Journal of biological chemistry.

[32]  B. Geiger,et al.  Enzyme replacement in Tay‐Sachs disease , 1979, Neurology.

[33]  N Hanai,et al.  Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases. , 1996, Human molecular genetics.