Gene transfer approaches to the lysosomal storage disorders.

Single-Step Method of RNA Isolation by Acid Guanidinium Thiocyanate–Phenol–Chloroform Extraction

1987 • 64,270 citations

‘Actively Acquired Tolerance’ of Foreign Cells

1953 • 3,103 citations

Perivascular Microglial Cells of the CNS Are Bone Marrow-Derived and Present Antigen in Vivo

1988 • 1,451 citations

Redesign of Retrovirus Packaging Cell Lines To Avoid Recombination Leading to Helper Virus Production

1986 • 1,276 citations

Grafts of Fetal Dopamine Neurons Survive and Improve Motor Function in Parkinson's Disease

1990 • 1,194 citations

Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

1994 • 1,032 citations

Multipotent neural cell lines can engraft and participate in development of mouse cerebellum

1992 • 928 citations

Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges.

1988 • 908 citations

Platelet-derived growth factor promotes division and motility and inhibits premature differentiation of the oligodendrocyte/type-2 astrocyte progenitor ceil

1988 • 833 citations

Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors

1996 • 747 citations

FGF modulates the PDGF-driven pathway of oligodendrocyte development

1990 • 586 citations

Cooperation between two growth factors promotes extended self-renewal and inhibits differentiation of oligodendrocyte-type-2 astrocyte (O-2A) progenitor cells.

1990 • 573 citations

Cytokine Stimulation of Multilineage Hematopoiesis from Immature Human Cells Engrafted in SCID Mice

1992 • 555 citations

Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

1996 • 542 citations

Human Fetal Dopamine Neurons Grafted Into the Striatum in Two Patients With Severe Parkinson's Disease

1989 • 516 citations

Neural progenitor cell engraftment corrects lysosomal storage throughout the MRS VII mouse brain

1995 • 475 citations

CYTOLOGICAL EVIDENCE FOR A RELATIONSHIP BETWEEN NORMAL HEMATOPOIETIC COLONY-FORMING CELLS AND CELLS OF THE LYMPHOID SYSTEM

1968 • 441 citations

Repair of demyelinated lesions by transplantation of purified 0-2A progenitor cells

1993 • 441 citations

Macrophages and the Nervous System

1991 • 429 citations

CG‐4, A new bipotential glial cell line from rat brain, is capable of differentiating in vitro into either mature oligodendrocytes or type‐2 astrocytes

1992 • 409 citations

Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA.

1994 • 379 citations

Insulin‐like growth factor I promotes cell proliferation and oligodendroglial commitment in rat glial progenitor cells developing in vitro

1988 • 361 citations

Transplantation of fetal dopamine neurons in Parkinson's disease: One‐year clinical and neurophysiological observations in two patients with putaminal implants

1992 • 360 citations

Proteoglycan-collagen arrangements in developing rat tail tendon. An electron microscopical and biochemical investigation

1981 • 348 citations

Quantitative correlation between the residual activity of β-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

1992 • 327 citations

A quantitative immunohistochemical study of macroglial cell development in the rat optic nerve: In vivo evidence for two distinct astrocyte lineages

1985 • 316 citations

Molecular Basis of Different Forms of Metachromatic Leukodystrophy

1991 • 291 citations

Differentiation of a bipotential glial progenitor cell in single cell microculture

1985 • 262 citations

Stimulation of oligodendroglial proliferation and maturation by interleukin-2

1986 • 251 citations

Cell Lines for the Production of Recombinant Adeno-Associated Virus

1995 • 250 citations

Phenotype of arylsulfatase A-deficient mice: Relationship to human metachromatic leukodystrophy

1996 • 242 citations

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

1992 • 221 citations

Cloning and Expression of Human Arylsulfatase A

1989 • 214 citations

Transplantation of fetal dopamine neurons in Parkinson's disease: PET {¹⁸F}6‐<scp>L</scp>‐fluorodopa studies in two patients with putaminal implants

1992 • 207 citations

Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

1992 • 196 citations

Long‐term outcome of Hurler syndrome following bone marrow transplantation

1993 • 192 citations

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease.

1994 • 190 citations

Centrifugal enhancement of retroviral mediated gene transfer

1995 • 189 citations

Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation

1991 • 186 citations

Male Donor-derived Cells in the Brains of Female Sex-mismatched Bone Marrow Transplant Recipients

1993 • 184 citations

Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

1993 • 183 citations

Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector.

1992 • 183 citations

Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells

1995 • 182 citations

Ovarian teratomas in mice are derived from oocytes that have completed the first meiotic division

1977 • 167 citations

Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation

1994 • 166 citations

Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.

1993 • 165 citations

Extracellular matrix-associated molecules collaborate with ciliary neurotrophic factor to induce type-2 astrocyte development.

1990 • 155 citations

Gaucher Disease in the Neonate: A Distinct Gaucher Phenotype Is Analogous to a Mouse Model Created by Targeted Disruption of the Glucocerebrosidase Gene

1992 • 149 citations

Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation

1990 • 149 citations

Stromal support enhances cell-free retroviral vector transduction of human bone marrow long-term culture-initiating cells

1992 • 141 citations

Transplantation of an oligodendrocyte cell line leading to extensive myelination.

1994 • 135 citations

Asymmetric replication in vitro from a human sequence element is dependent on adeno-associated virus Rep protein

1995 • 130 citations

Structure of the arylsulfatase A gene

1990 • 123 citations

Determination of the origin and nature of brain macrophages and microglial cells in mouse central nervous system, using non‐radioactive in situ hybridization and immunoperoxidase techniques

1992 • 122 citations

High-Efficiency Transfer of the T Cell Co-Stimulatory Molecule B7-2 to Lymphoid Cells Using High-Titer Recombinant Adeno-Associated Virus Vectors

1995 • 109 citations

Pluripotential hemopoietic stem cells in adult mouse brain.

1982 • 106 citations

Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products

1997 • 103 citations

Expansion of rat oligodendrocyte progenitors into proliferative ?oligospheres? that retain differentiation potential

1996 • 100 citations

Recombinant junctions formed by site-specific integration of adeno-associated virus into an episome

1995 • 93 citations

Type 2 and type 3 Gaucher disease: A morphological and biochemical study

1986 • 91 citations

Ontogeny of human hematopoietic cells: analysis utilizing monoclonal antibodies.

1983 • 91 citations

Antisense Inhibition and Adeno-Associated Viral Vector Delivery for Reducing Hypertension

1997 • 90 citations

Fetal Homotransplants (Ventral Mesencephalon and Adrenal Tissue) to the Striatum of Parkinsonian Subjects

1990 • 88 citations

BONE-MARROW TRANSPLANTATION FOR METACHROMATIC LEUCODYSTROPHY

1985 • 82 citations

Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells

1993 • 82 citations

Growth factors and stromal support generate very efficient retroviral transduction of peripheral blood CD34+ cells from Gaucher patients

1995 • 78 citations

Transplantation of Human Embryonic Oligodendrocytes into Shiverer Brain^a

1987 • 78 citations

Bone marrow transplantation in canine mucopolysaccharidosis I. Effects within the central nervous system.

1987 • 77 citations

Ultrastructural studies of eight cases of fetal Tay-Sachs disease.

1974 • 77 citations

Bone Marrow and Lymphoid Cell Injection of the Pig Foetus resulting in Transplantation Tolerance or Immunity, and Immunoglobulin Production

1967 • 76 citations

Tracing transplanted oligodendrocytes during migration and maturation in the shiverer mouse brain

1991 • 72 citations

Transplantations of Newborn CNS Fragments into the Brain of Shiverer Mutant Mice: Extensive Myelination by Transplanted Oligodendrocytes

1986 • 68 citations

Long-term survival of grafted cells, dopamine synthesis/release, synaptic connections, and functional recovery after transplantation of fetal nigral cells in rats with unilateral 6-OHDA lesions in the nigrostriatal dopamine pathway

1990 • 66 citations

Bone marrow transplantation for Sanfilippo disease type B

1992 • 65 citations

Myelin formation following transplantation of normal fetal glia into myelin-deficient rat spinal cord

1990 • 65 citations

High-Level Globin Gene Expression Mediated by a Recombinant Adeno-Associated Virus Genome That Contains the 3′ γ Globin Gene Regulatory Element and Integrates as Tandem Copies in Erythroid Cells

1997 • 64 citations

Infantile (type II) Gaucher's disease: In utero diagnosis and fetal pathology

1972 • 61 citations

CNS neuronal cell line-derived factors regulate gliogenesis in neonatal rat brain cultures

1988 • 60 citations

Determination of adeno-associated virus Rep68 and Rep78 binding sites by random sequence oligonucleotide selection

1995 • 57 citations

Delivery of active hexosaminidase across the blood‐brain barrier in rats

1984 • 56 citations

Efficient retroviral gene transfer to purified long-term repopulating hematopoietic stem cells

1994 • 55 citations

Retroviral vector design for long-term expression in murine hematopoietic cells in vivo

1994 • 53 citations

Transplanted Transgenically Marked Oligodendrocytes Survive, Migrate and Myelinate in the Normal Mouse Brain as They Do in the Shiverer Mouse Brain

1994 • 52 citations

Chapter 83 Neural transplantation in Parkinson's disease: the Swedish experience

1990 • 51 citations

Transplantation of oligodendrocytes in the newborn mouse brain: extension of myelination by transplanted cells. Anatomical study

1987 • 50 citations

Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.

1994 • 49 citations

Retroviral Stem Cell Gene Therapy

1997 • 48 citations

Retroviral vector-mediated transfer of the bacterial neomycin resistance gene into fetal and adult sheep and human hematopoietic progenitors in vitro

1990 • 47 citations

Bone marrow transplantation in the treatment of alpha-mannosidosis.

1987 • 43 citations

Amphetamine-induced behavioral phenotype in a hypoxanthine^guanine phosphoribosyltransferase-deficient mouse model of Lesch-Nyhan syndrome.

1991 • 43 citations

Long-Term Neurological Effects of Bone Marrow Transplantation in a Canine Lysosomal Storage Disease

1988 • 40 citations

Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review.

1994 • 39 citations

Myelination by oligodendrocytes isolated from 4–6-week-old rat central nervous system and transplanted into newborn shiverer brain

1988 • 39 citations

Molecular genetics of metachromatic leukodystrophy

1994 • 39 citations

Fetal liver transplantation: biology and clinical results.

1993 • 33 citations

Myelination by Cryopreserved Xenografts and Allografts in the Myelin-Deficient Rat

1994 • 32 citations

Structure of the Mouse Arylsulfatase A Gene and cDNA

1994 • 30 citations

Increased titer of recombinant AAV vectors by gene transfer with adenovirus coupled to DNA-polylysine complexes.

1995 • 27 citations

Expression of the human glucocerebrosidase and arylsulfatase A genes in murine and patient primary fibroblasts transduced by an adeno-associated virus vector.

1994 • 27 citations

Gene Transfer into Hematopoietic Cells: Implications for Cancer Therapy

1994 • 27 citations

Chimerism Following Fetal Transfusion

1973 • 26 citations

Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation

1994 • 25 citations

Retroviral gene transfer and sustained expression of human arylsulfatase A.

1996 • 24 citations

A further example of human blood group chimaerism.

1977 • 17 citations

Efficient retroviral mediated transfer of the glucocerebrosidase gene in CD34+ enriched umbilical cord blood human hematopoietic progenitors.

1995 • 15 citations

Correction of Murine Mucopolysaccharidosis Type VII (MPS VII) by Bone Marrow Transplantation and Gene Transfer Therapy

1991 • 14 citations

Lysosomal enzyme replacement in neural tissue by allogeneic bone marrow transplantation following total lymphoid irradiation in canine fucosidosis.

1987 • 14 citations

Fetal Metachromatic Leukodystrophy: Pathology, Biochemistry and a Study of In Vitro Enzyme Replacement in CNS Tissue

1976 • 13 citations

Histological improvement and enzyme replacement in the brains of fucosidosis dogs after bone marrow engraftment.

1989 • 13 citations

Quantitative aspects of fetal liver cell transplantation in animals and man.

1976 • 11 citations

Correction of enzyme deficiency in metachromatic leukodystrophy fibroblasts by retroviral‐mediated transfer of the human arylsulphtase A gene

1993 • 11 citations

Transduction, Expression, and Secretion of Human Glucocerebrosidase by Murine Myoblasts

1994 • 7 citations

The O-2A Lineage: From Rats to Humans

1994 • 6 citations

Sulfatides in Prenatal Metachromatic Leukodystrophy

1983 • 6 citations

SCID-hu mice as a model to study tolerance after fetal stem cell transplantation.

1992 • 5 citations