Published in Cell on August 01, 1980
RNA processing errors in patients with beta-thalassemia. Proc Natl Acad Sci U S A (1982) 2.99
Direct identification of sickle cell anemia by blot hybridization. Proc Natl Acad Sci U S A (1981) 2.68
Base substitution in an intervening sequence of a beta+-thalassemic human globin gene. Proc Natl Acad Sci U S A (1981) 2.58
Defects in RNA splicing and the consequence of shortened translational reading frames. Am J Hum Genet (1996) 2.45
beta zero thalassemia in Sardinia is caused by a nonsense mutation. J Clin Invest (1981) 2.30
An enhancer element lies 3' to the human A gamma globin gene. EMBO J (1987) 2.11
Replication and expression of thymidine kinase and human globin genes microinjected into mouse fibroblasts. Proc Natl Acad Sci U S A (1980) 2.10
A nucleotide change at a splice junction in the human beta-globin gene is associated with beta 0-thalassemia. Proc Natl Acad Sci U S A (1981) 1.93
An intron nucleotide sequence variant in a cloned beta +-thalassaemia globin gene. Nucleic Acids Res (1981) 1.80
mRNA-deficient beta o-thalassemia results from a single nucleotide deletion. Nucleic Acids Res (1982) 1.78
Accurate in vitro splicing of human beta-globin RNA. Nucleic Acids Res (1982) 1.73
Transforming growth factor beta reverses the glucocorticoid-induced wound-healing deficit in rats: possible regulation in macrophages by platelet-derived growth factor. Proc Natl Acad Sci U S A (1989) 1.73
Mutation in an intervening sequence splice junction in man. Proc Natl Acad Sci U S A (1981) 1.34
"Silent" nucleotide substitution in a beta+-thalassemia globin gene activates splice site in coding sequence RNA. Proc Natl Acad Sci U S A (1983) 1.25
Characterization of the porphobilinogen deaminase deficiency in acute intermittent porphyria. Immunologic evidence for heterogeneity of the genetic defect. J Clin Invest (1981) 1.22
The thalassemias: molecular mechanisms of human genetic disease. Am J Hum Genet (1983) 1.21
Transfer of nonselectable genes into mouse teratocarcinoma cells and transcription of the transferred human beta-globin gene. Mol Cell Biol (1982) 1.19
Activation of the human beta-globin promoter in K562 cells by DNA sequences 5' to the fetal gamma- or embryonic zeta-globin genes. J Clin Invest (1987) 1.19
Presence of albumin mRNA precursors in nuclei of analbuminemic rat liver lacking cytoplasmic albumin mRNA. Proc Natl Acad Sci U S A (1982) 1.17
A splice junction deletion deficient in the transport of RNA does not polyadenylate nuclear RNA. Mol Cell Biol (1983) 1.16
Two cloned beta thalassemia genes are associated with amber mutations at codon 39. Nucleic Acids Res (1981) 1.14
Beta-thalassemia resulting from a single nucleotide substitution in an acceptor splice site. Nucleic Acids Res (1985) 1.09
Loss of a consensus splice signal in a mutant immunoglobulin gene eliminates the CH1 domain exon from the mRNA. Mol Cell Biol (1984) 1.09
Molecular analysis of the beta-thalassemia phenotype associated with inheritance of hemoglobin E (alpha 2 beta2(26)Glu leads to Lys). J Clin Invest (1981) 1.04
5-Azacytidine acts directly on both erythroid precursors and progenitors to increase production of fetal hemoglobin. J Clin Invest (1985) 1.00
Developmental programs of human erythroleukemia cells: globin gene expression and methylation. Mol Cell Biol (1988) 0.92
Posttranscriptional defects in beta-globin messenger RNA metabolism in beta-thalassemia: abnormal accumulation of beta-messenger RNA precursor sequences. J Clin Invest (1981) 0.90
Five nucleotide changes in the large intervening sequence of a beta globin gene in a beta+ thalassemia patient. Nucleic Acids Res (1982) 0.90
Genetic test for involvement of intervening sequences in transport of nuclear RNA. Mol Cell Biol (1982) 0.89
Restriction fragment length polymorphism of the rat albumin gene in Sprague-Dawley rats and its application in genetic study of analbuminemia. Nucleic Acids Res (1982) 0.86
Altered globin gene transcription pattern and the presence of a 7-8 kb alpha A globin gene transcript in avian erythroblastosis virus-transformed cells. EMBO J (1984) 0.77
beta (+)-Thalassaemia in the Po river delta region (northern Italy): genotype and beta globin synthesis. J Med Genet (1985) 0.76
Transfer of human and murine globin-gene sequences into transgenic mice. Am J Hum Genet (1985) 0.75
Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N Engl J Med (1994) 4.86
An oligomer complementary to c-myc mRNA inhibits proliferation of HL-60 promyelocytic cells and induces differentiation. Mol Cell Biol (1988) 4.08
Tandem AP-1-binding sites within the human beta-globin dominant control region function as an inducible enhancer in erythroid cells. Genes Dev (1990) 4.07
A family of long reiterated DNA sequences, one copy of which is next to the human beta globin gene. Nucleic Acids Res (1980) 3.91
Inducible production of c-fos antisense RNA inhibits 3T3 cell proliferation. Proc Natl Acad Sci U S A (1986) 3.71
5-azacytidine selectively increases gamma-globin synthesis in a patient with beta+ thalassemia. N Engl J Med (1982) 3.29
The functional human dihydrofolate reductase gene. J Biol Chem (1984) 3.28
RNA processing errors in patients with beta-thalassemia. Proc Natl Acad Sci U S A (1982) 2.99
Chromosomal organization of the human dihydrofolate reductase genes: dispersion, selective amplification, and a novel form of polymorphism. Proc Natl Acad Sci U S A (1984) 2.99
Differences in human alpha-, beta- and delta-globin gene expression in monkey kidney cells. Cell (1982) 2.78
Augmentation by erythropoietin of the fetal-hemoglobin response to hydroxyurea in sickle cell disease. N Engl J Med (1993) 2.50
Dysregulated interleukin 6 expression produces a syndrome resembling Castleman's disease in mice. J Clin Invest (1990) 2.37
Continuous subcutaenous administration of deferoxamine in patients with iron overload. N Engl J Med (1977) 2.31
Expression of the human c-fms proto-oncogene in hematopoietic cells and its deletion in the 5q- syndrome. Cell (1985) 2.28
Beta O-39 thalassemia gene: a premature termination codon causes beta-mRNA deficiency without affecting cytoplasmic beta-mRNA stability. Blood (1984) 2.17
Developmental regulation of human globin genes. Annu Rev Biochem (1985) 2.16
Enforced expression of the GATA-2 transcription factor blocks normal hematopoiesis. Blood (1999) 2.14
Purification of the human NF-E2 complex: cDNA cloning of the hematopoietic cell-specific subunit and evidence for an associated partner. Mol Cell Biol (1993) 2.05
Cloning and characterization of DNA sequences surrounding the human gamma-, delta-, and beta-globin genes. Proc Natl Acad Sci U S A (1980) 2.04
Inducibility of the HS II enhancer depends on binding of an erythroid specific nuclear protein. Nucleic Acids Res (1990) 2.01
Pure red-cell aplasia of 10 years' duration due to persistent parvovirus B19 infection and its cure with immunoglobulin therapy. N Engl J Med (1989) 1.85
Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sci U S A (1989) 1.84
Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector. Proc Natl Acad Sci U S A (1992) 1.82
Expression of the human c-fms proto-oncogene product (colony-stimulating factor-1 receptor) on peripheral blood mononuclear cells and choriocarcinoma cell lines. J Clin Invest (1986) 1.77
Hepatic iron stores and plasma ferritin concentration in patients with sickle cell anemia and thalassemia major. Am J Hematol (1993) 1.76
Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood (1994) 1.75
Translation of rabbit haemoglobin messenger RNA by thalassaemic and non-thalassaemic ribosomes. Nat New Biol (1971) 1.75
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood (2001) 1.74
Isolation and translation of hemoglobin messenger RNA from thalassemia, sickle cell anemia, and normal human reticulocytes. J Clin Invest (1971) 1.74
Intronless human dihydrofolate reductase genes are derived from processed RNA molecules. Proc Natl Acad Sci U S A (1982) 1.69
Mechanism of DNase I hypersensitive site formation within the human globin locus control region. Proc Natl Acad Sci U S A (1992) 1.67
The interleukin 3 gene is located on human chromosome 5 and is deleted in myeloid leukemias with a deletion of 5q. Proc Natl Acad Sci U S A (1987) 1.64
Targeted and highly efficient gene transfer into CD4+ cells by a recombinant human immunodeficiency virus retroviral vector. J Clin Invest (1991) 1.64
Active conformation of the globin genes in uninduced and induced mouse erythroleukemia cells. Cell (1978) 1.64
Hematologic responses of patients with sickle cell disease to treatment with hydroxyurea. N Engl J Med (1990) 1.63
Purification and characterization of heterogeneous pluripotent hematopoietic stem cell populations expressing high levels of c-kit receptor. Blood (1993) 1.62
5-Azacytidine increases gamma-globin synthesis and reduces the proportion of dense cells in patients with sickle cell anemia. Blood (1983) 1.61
Transduction and expression of the human carcinoembryonic antigen gene in a murine colon carcinoma cell line. Cancer Res (1991) 1.60
Development of a high-titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells. Proc Natl Acad Sci U S A (1990) 1.59
Selective activation of human beta-but not gamma-globin gene in human fibroblast x mouse erythroleukaemia cell hybrids. Nature (1979) 1.58
Cyclosporine therapy of aplastic anaemia, congenital and acquired red cell aplasia. Br J Haematol (1989) 1.56
Cell-free transcription of mammalian chromatin: transcription of globin messenger RNA sequences from bone-marrow chromatin with mammalian RNA polymerase. Proc Natl Acad Sci U S A (1974) 1.54
Isolation and characterization of a 15-kilobase genomic sequence coding for part of the Pro alpha 2 chain of sheep type I collagen. J Biol Chem (1980) 1.53
Advances in thalassemia research. Blood (1984) 1.52
Treatment of Diamond-Blackfan anaemia with haematopoietic growth factors, granulocyte-macrophage colony stimulating factor and interleukin 3: sustained remissions following IL-3. Br J Haematol (1991) 1.50
Hydroxyurea-induced HbF production in anemic primates: augmentation by erythropoietin, hematopoietic growth factors, and sodium butyrate. Exp Hematol (1992) 1.50
Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science (1992) 1.49
Brief report: treatment with azacitidine of patients with end-stage beta-thalassemia. N Engl J Med (1993) 1.48
Promoter sequences required for function of the human gamma globin gene in erythroid cells. EMBO J (1986) 1.47
Contribution of promoter to tissue-specific expression of the mouse immunoglobulin kappa gene. Science (1985) 1.44
Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest (1994) 1.39
An array of murine leukemia virus-related elements is transmitted and expressed in a primate recipient of retroviral gene transfer. J Virol (1996) 1.38
Inducible, high-level production of infectious murine leukemia retroviral vector particles pseudotyped with vesicular stomatitis virus G envelope protein. Hum Gene Ther (1995) 1.37
Relative stability of alpha- and beta-globin messenger RNAs in homozygous beta+ thalassemia. Proc Natl Acad Sci U S A (1977) 1.36
Expression of the human beta-globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice. Proc Natl Acad Sci U S A (1988) 1.36
Human globin gene promoter sequences are sufficient for specific expression of a hybrid gene transfected into tissue culture cells. Mol Cell Biol (1987) 1.35
Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein. Proc Natl Acad Sci U S A (1987) 1.34
Retrovirus-mediated transfer and expression of the interleukin-3 gene in mouse hematopoietic cells result in a myeloproliferative disorder. Mol Cell Biol (1989) 1.34
Recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) shortens the period of neutropenia after autologous bone marrow transplantation in a primate model. J Clin Invest (1987) 1.33
Periodic microcirculatory flow in patients with sickle-cell disease. N Engl J Med (1984) 1.32
Hemoglobin messenger RNA from human bone marrow. Isolation and translation in homozygous and heterozygous beta-thalassemia. J Clin Invest (1973) 1.32
Hemoglobin switching in man and chicken is mediated by a heteromeric complex between the ubiquitous transcription factor CP2 and a developmentally specific protein. EMBO J (1995) 1.30
Evidence of genetic heterogeneity in five kindreds with familial hypertrophic cardiomyopathy. Circulation (1992) 1.29
Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells. Proc Natl Acad Sci U S A (1994) 1.28
Regulation of hemoglobin synthesis during the development of the red cell (third of three parts). N Engl J Med (1977) 1.28
An olfactory receptor gene is located in the extended human beta-globin gene cluster and is expressed in erythroid cells. Genomics (1999) 1.28
Cell-free hemoglobin synthesis in beta-thalassemia. Proc Natl Acad Sci U S A (1970) 1.28
Characterization of replication-competent retroviruses from nonhuman primates with virus-induced T-cell lymphomas and observations regarding the mechanism of oncogenesis. J Virol (1994) 1.28
COOH-terminal-modified interleukin-3 is retained intracellularly and stimulates autocrine growth. Science (1989) 1.27
Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy. Blood (2001) 1.26
"Silent" nucleotide substitution in a beta+-thalassemia globin gene activates splice site in coding sequence RNA. Proc Natl Acad Sci U S A (1983) 1.25
Diversified prime and boost protocols using recombinant vaccinia virus and recombinant non-replicating avian pox virus to enhance T-cell immunity and antitumor responses. Vaccine (1997) 1.24
Therapeutic approaches to hemoglobin switching in treatment of hemoglobinopathies. Annu Rev Med (1992) 1.24
Purification and properties of rabbit reticulocyte protein synthesis elongation factor 2. J Biol Chem (1975) 1.21
Retroviral transfer and expression of the interleukin-3 gene in hemopoietic cells. Genes Dev (1987) 1.21
A beta-globin gene, inactive in the K562 leukemic cell, functions normally in a heterologous expression system. Proc Natl Acad Sci U S A (1984) 1.21
A weak upstream promoter gives rise to long human beta-globin RNA molecules. Biochem Biophys Res Commun (1983) 1.20
Vitamin C and iron. N Engl J Med (1981) 1.19
Bone marrow-derived cells contribute to tumor neovasculature and, when modified to express an angiogenesis inhibitor, can restrict tumor growth in mice. Clin Cancer Res (2001) 1.19
Activation of the human beta-globin promoter in K562 cells by DNA sequences 5' to the fetal gamma- or embryonic zeta-globin genes. J Clin Invest (1987) 1.19
Dihydrofolate reductase gene amplification and possible rearrangement in estrogen-responsive methotrexate-resistant human breast cancer cells. J Biol Chem (1982) 1.17
Gene therapy for human hemoglobinopathies. Proc Soc Exp Biol Med (1993) 1.15
Transfusion haemosiderosis and chelation therapy. Clin Haematol (1982) 1.15
Site-specific demethylation and normal chromatin structure of the human dihydrofolate reductase gene promoter after transfection into CHO cells. Mol Cell Biol (1987) 1.13
Stable gene transfer and tissue-specific expression of a human globin gene using adenoviral vectors. EMBO J (1986) 1.13
An improved method for generating retroviral producer clones for vectors lacking a selectable marker gene. Blood Cells Mol Dis (1998) 1.12
Only the promoter region of the constitutively expressed normal and amplified human dihydrofolate reductase gene is DNase I hypersensitive and undermethylated. J Biol Chem (1985) 1.12
Transfer of genes into hematopoietic cells using recombinant DNA viruses. Proc Natl Acad Sci U S A (1985) 1.12