Developments Leading to Human Gene Therapy

The Metabolic Basis of Inherited Disease.

1988 • 7,933 citations

Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus

1983 • 1,857 citations

Stable replication of plasmids derived from Epstein–Barr virus in various mammalian cells

1985 • 1,303 citations

Human GM-CSF: Molecular Cloning of the Complementary DNA and Purification of the Natural and Recombinant Proteins

1985 • 1,253 citations

Construction and applications of a highly transmissible murine retrovirus shuttle vector

1984 • 1,020 citations

Enhancer-dependent expression of human kappa immunoglobulin genes introduced into mouse pre-B lymphocytes by electroporation.

1984 • 889 citations

RNA tumor viruses

1982 • 833 citations

Structures of P transposable elements and their sites of insertion and excision in the Drosophila melanogaster genome

1983 • 794 citations

Genetic engineering. Principles and methods

1987 • 715 citations

Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hemopoietic system of W/W mice

1985 • 663 citations

Vaccinia virus: a selectable eukaryotic cloning and expression vector.

1982 • 663 citations

General method for production and selection of infectious vaccinia virus recombinants expressing foreign genes

1984 • 652 citations

Carbonic anhydrase II deficiency identified as the primary defect in the autosomal recessive syndrome of osteopetrosis with renal tubular acidosis and cerebral calcification.

1983 • 609 citations

Prospects for Human Gene Therapy

1984 • 548 citations

Medicine and the Law

1987 • 541 citations

Characterization of a “silencer” in yeast: A DNA sequence with properties opposite to those of a transcriptional enhancer

1985 • 531 citations

Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse

1984 • 527 citations

A third type of murine T-cell receptor gene

1984 • 491 citations

REVERSAL OF CLINICAL FEATURES OF HURLER'S DISEASE AND BIOCHEMICAL IMPROVEMENT AFTER TREATMENT BY BONE-MARROW TRANSPLANTATION

1981 • 490 citations

Genetic and physical linkage of exogenous sequences in transformed cells

1980 • 486 citations

Specific cloning of DNA fragments absent from the DNA of a male patient with an X chromosome deletion.

1985 • 455 citations

Successful Bone-Marrow Transplantation for Infantile Malignant Osteopetrosis

1980 • 449 citations

Liver Transplantation to Provide Low-Density-Lipoprotein Receptors and Lower Plasma Cholesterol in a Child with Homozygous Familial Hypercholesterolemia

1984 • 442 citations

DOUBLE-BLIND COMPARISON OF CAPTOPRIL ALONE AGAINST FRUSEMIDE PLUS AMILORIDE IN MILD HEART FAILURE

1987 • 429 citations

Use of synthetic oligonucleotides as hybridization probes: isolation of cloned cDNA sequences for human beta 2-microglobulin.

1981 • 423 citations

High-efficiency gene transfer into mammalian cells: generation of helper-free recombinant retrovirus with broad mammalian host range.

1984 • 407 citations

Transformation of white locus DNA in Drosophila: Dosage compensation, zeste interaction, and position effects

1984 • 388 citations

The effect of chromosomal position on the expression of the drosophila xanthine dehydrogenase gene

1983 • 368 citations

Chromosomal position and activation of retroviral genomes inserted into the germ line of mice

1981 • 344 citations

Generation of helper-free amphotropic retroviruses that transduce a dominant-acting, methotrexate-resistant dihydrofolate reductase gene.

1985 • 342 citations

MARROW TRANSPLANTATION FOR THALASSAEMIA

1982 • 330 citations

Adenosine deaminase: characterization and expression of a gene with a remarkable promoter.

1985 • 329 citations

PROLONGED PERITONEAL DIALYSIS FOR CHRONIC RENAL FAILURE

1964 • 328 citations

Carbonic Anhydrase II Deficiency in 12 Families with the Autosomal Recessive Syndrome of Osteopetrosis with Renal Tubular Acidosis and Cerebral Calcification

1985 • 322 citations

Determination of the leukaemogenicity of a murine retrovirus by sequences within the long terminal repeat

1984 • 321 citations

The Natural History of the Inherited Methylmalonic Acidemias

1983 • 317 citations

Bone-Marrow Transplantation in a Patient with Sickle-Cell Anemia

1984 • 306 citations

Coamplification and Coexpression of Human Tissue-Type Plasminogen Activator and Murine Dihydrofolate Reductase Sequences in Chinese Hamster Ovary Cells

1985 • 269 citations

Complete Correction of the Wiskott-Aldrich Syndrome by Allogeneic Bone-Marrow Transplantation

1978 • 256 citations

Retroviruses as mutagens: Insertion and excision of a nontransforming provirus alter expression of a resident transforming provirus

1981 • 253 citations

Prenatal Genetic Diagnosis in 2,000 Amniocenteses

1999 • 231 citations

THE MUTATION RATE OF THE GENE FOR HAEMOPHILIA, AND ITS SEGREGATION RATIOS IN MALES AND FEMALES

1946 • 226 citations

USE OF ANTI-T-CELL MONOCLONAL ANTIBODY OKT3 TO PREVENT ACUTE GRAFT-VERSUS-HOST DISEASE IN ALLOGENEIC BONE-MARROW TRANSPLANTATION FOR ACUTE LEUKAEMIA

1982 • 220 citations

Human Liver Transplantation: Analysis of Data on 540 Patients from Four Centers

1984 • 218 citations

Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells

1983 • 214 citations

Coamplification and coexpression of human tissue-type plasminogen activator and murine dihydrofolate reductase sequences in Chinese hamster ovary cells.

1985 • 208 citations

A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): gene transfer into cells obtained from humans deficient in HPRT.

1983 • 208 citations

Enhancing the efficiency of DNA-mediated gene transfer in mammalian cells

1981 • 208 citations

Bone-Marrow Transplantation in the Maroteaux–Lamy Syndrome (Mucopolysaccharidosis Type VI)

1984 • 203 citations

Infection of preimplantation mouse embryos and of newborn mice with leukemia virus: tissue distribution of viral DNA and RNA and leukemogenesis in the adult animal.

1975 • 198 citations

Epstein–Barr Virus–Associated B-Cell Proliferations of Diverse Clonal Origins after Bone Marrow Transplantation in a 12-Year-Old Patient with Severe Combined Immunodeficiency

1985 • 198 citations

Expression of a Retrovirus Encoding Human HPRT in Mice

1984 • 182 citations

Bovine papillomavirus vector that propagates as a plasmid in both mouse and bacterial cells.

1982 • 180 citations

Severe combined immunodeficiency disease. Characterization of the disease and results of transplantation.

1977 • 177 citations

Isolation of the human insulin-like growth factor I gene using a single synthetic DNA probe.

1984 • 177 citations

Inherited disorder of purine metabolism. Correlation between central nervous system dysfunction and biochemical defects

1967 • 172 citations

Amyloid macroglossia; report of a case.

1947 • 162 citations

Immunological screening method to detect specific translation products.

1978 • 160 citations

Transformation of mammalian cells with an amplifiable dominant-acting gene.

1980 • 158 citations

Immunologic reconstitution after human marrow grafting.

1984 • 154 citations

Hepatic Adenomata With Type 1 Glycogen Storage Disease

1976 • 142 citations

Insertion Mutagenesis of Embryonal Carcinoma Cells by Retroviruses

1985 • 132 citations

Inherited Disorder of Purine Metabolism

1967 • 130 citations

Homologous recombination in mammalian cells mediates formation of a functional gene from two overlapping gene fragments.

1983 • 129 citations

Genetic control of cellular susceptibility to pseudotypes of rous sarcoma virus

1965 • 127 citations

Efficient insertion of genes into the mouse germ line via retroviral vectors.

1985 • 126 citations

Late complications after marrow transplantation.

1984 • 123 citations

Transformation mediated by the SV40 T antigens: Separation of the overlapping SV40 early genes with a retroviral vector

1984 • 121 citations

Expression of genes introduced into cells by retroviral infection is more efficient than that of genes introduced into cells by DNA transfection

1984 • 121 citations

Experimental manipulation of gene expression

1983 • 115 citations

Correction of infantile agranulocytosis (Kostmann's syndrome) by allogeneic bone marrow transplantation

1980 • 107 citations

The Metabolic Basis of Inherited Disease

1983 • 105 citations

Severe Combined Immunodeficiency Disease

1977 • 104 citations

Malignant Transformation of Erythroid Cells in Vivo by Introduction of a Nonreplicating Retrovirus Vector

1985 • 103 citations

Orthotopic Liver Transplantation

2008 • 102 citations

Marrow transplantation for congenital disorders.

1984 • 102 citations

Marrow transplantation for juvenile osteopetrosis

1981 • 98 citations

Bone marrow transplantation only partially restores purine metabolites to normal in adenosine deaminase-deficient patients.

1981 • 97 citations

Partial phenotypic correction of human Lesch-Nyhan (hypoxanthine-guanine phosphoribosyltransferase-deficient) lymphoblasts with a transmissible retroviral vector.

1984 • 96 citations

High-frequency deletion in recovered retrovirus vectors containing exogenous DNA with promoters

1984 • 96 citations

Introduction of a selectable gene into different animal tissue by a retrovirus recombinant vector.

1984 • 88 citations

Correction of feline arylsulphatase B deficiency (mucopolysaccharidosis VI) by bone marrow transplantation

1984 • 86 citations

Marrow transplantation for thalassemia.

1986 • 84 citations

Cloning of cDNA sequences of human adenosine deaminase.

1983 • 83 citations

Efficient transformation of human fibroblasts by adenovirus-simian virus 40 recombinants.

1984 • 80 citations

The early complications of bone marrow transplantation.

1984 • 80 citations

Prenatal diagnosis of genetic disorders.

1976 • 78 citations

Analysis of Liver Transplantation

1984 • 76 citations

Selective overproduction of adenosine deaminase in cultured mouse cells.

1983 • 75 citations

Molecular cloning of human adenosine deaminase gene sequences.

1983 • 71 citations

Bone marrow transplantation for Fanconi anemia

1995 • 68 citations

SIMIAN VIRUS 40 AS A EUKARYOTIC CLONING VEHICLE

1981 • 63 citations

Utilization of prenatal genetic diagnosis in women 35 years of age and older in the United States, 1977 to 1978

1981 • 62 citations

Retrovirus transduction: generation of infectious retroviruses expressing dominant and selectable genes is associated with in vivo recombination and deletion events.

1983 • 61 citations

No apparent nucleotide sequence specificity in cellular DNA juxtaposed to retrovirus proviruses.

1980 • 61 citations

Structure of adenosine deaminase mRNAs from normal and adenosine deaminase-deficient human cell lines.

1984 • 60 citations

The Cambridge and King's College Hospital Experience of Liver Transplantation, 1968-1983

1984 • 60 citations

Isolation of cDNA clones for human adenosine deaminase

1983 • 58 citations

Transfer of genes into hematopoietic cells using recombinant DNA viruses.

1985 • 58 citations

Retroviral-mediated gene transfer of human phenylalanine hydroxylase into NIH 3T3 and hepatoma cells.

1986 • 55 citations

Rapid assay for extrachromosomal homologous recombination in monkey cells.

1985 • 55 citations

Recombination of transfected DNAs in vertebrate cells in culture.

1984 • 55 citations

Inactivation of an acceptor RNA splice site by a short deletion in beta-thalassemia.

1983 • 54 citations

Allogeneic bone marrow transplantation for chronic granulomatous disease

1982 • 54 citations

Expression of complete chicken thymidine kinase gene inserted in a retrovirus vector.

1984 • 51 citations

Amplification and molecular cloning of murine adenosine deaminase gene sequences.

1983 • 48 citations

Retrovirus transduction: segregation of the viral transforming function and the herpes simplex virus tk gene in infectious Friend spleen focus-forming virus thymidine kinase vectors.

1983 • 48 citations

BONE MARROW TRANSPLANTATION IN THE WISKOTT-ALDRICH SYNDROME

1982 • 38 citations

Epidemiology and Clinical Course of Liver Diseases: Identification of Candidates for Hepatic Transplantation

1984 • 38 citations

Liver transplantation: the recent Cambridge-King's College Hospital experience.

1987 • 38 citations

Expression of human adenosine deaminase using a transmissable murine retrovirus vector system.

1985 • 36 citations

Conditioning regimens for marrow grafting.

1984 • 34 citations

Construction of Highly Transmissible Mammalian Cloning Vehicles Derived from Murine Retroviruses

1983 • 31 citations

Five-Year Survival After Liver Transplantation.

1985 • 31 citations

The monomeric glutamyl-tRNA synthetase from Bacillus subtilis 168 and its regulatory factor. Their purification, characterization, and the study of their interaction.

1983 • 28 citations

Nitrogen Metabolism in Neonatal Citrullinaemia

1977 • 28 citations

Increased expression of one of two adenosine deaminase alleles in a human choriocarcinoma cell line following selection with adenine nucleosides.

1983 • 26 citations

Prenatal genetic diagnosis and elective abortion in women over 35: Utilization and relative impact on the birth prevalence of Down syndrome in Washington State

1980 • 26 citations

Introduction of sequences encoding functional human adenosine deaminase into mouse cells using a retroviral shuttle system

1985 • 24 citations

Competence for DNA transfer of ouabain resistance and thymidine kinase: Clonal variation in mouse L-cell recipients

1981 • 24 citations

Gene Transfer with Retrovirus Vectors

1985 • 22 citations

Transplantation of hematopoietic cells for lethal congenital immunodeficiencies.

1983 • 20 citations

Bibliography of cloned human and other selected DNAs.

1985 • 18 citations

Mucopolysaccharidosis VI (Maroteaux-Lamy disease). Clinical and biochemical study of a mild variant case.

1974 • 17 citations

Genetic engineering of mammalian cells: its potential application to genetic diseases of man.

1982 • 11 citations

Retrovirus-mediated Gene Transfer of Argininosuccinate Synthetase into Cultured Rodent Cells and Human Citrullinemic Fibroblasts

1986 • 11 citations

Gene Therapy

1981 • 7 citations

Use of Retrovirus-Derived Vectors to Introduce and Express Genes in Mammalian Cells

1983 • 5 citations

Clinical and experimental transplantation in enzymatic deficiency disease.

1978 • 5 citations