Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin

A Third-Generation Lentivirus Vector with a Conditional Packaging System

1998 • 3,175 citations

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

1997 • 1,977 citations

Position-independent, high-level expression of the human β-globin gene in transgenic mice

1987 • 1,955 citations

The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA

1989 • 1,280 citations

Hematology of Infancy and Childhood.

1975 • 918 citations

Transduction of Human CD34 ⁺ Cells That Mediate Long-Term Engraftment of NOD/SCID Mice by HIV Vectors

1999 • 648 citations

The Molecular Basis of Blood Diseases.

1987 • 648 citations

The "beta-like-globin" gene domain in human erythroid cells.

1985 • 634 citations

Thalassemia Syndromes

2009 • 476 citations

Looping versus linking: toward a model for long-distance gene activation

1999 • 465 citations

Evidence for a locus activation region: the formation of developmentally stable hypersensitive sites in globin-expressing hybrids

1987 • 440 citations

The Molecular Basis of Blood Diseases

1994 • 370 citations

A dominant control region from the human β-globin locus conferring integration site-independent gene expression

1989 • 346 citations

Hematology of Infancy and Childhood

1993 • 283 citations

A mouse model for beta 0-thalassemia.

1995 • 262 citations

Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene.

1995 • 201 citations

The cHS4 Insulator Increases the Probability of Retroviral Expression at Random Chromosomal Integration Sites

2000 • 197 citations

Synergistic regulation of human beta-globin gene switching by locus control region elements HS3 and HS4.

1995 • 190 citations

Looping, Linking, and Chromatin Activity

2000 • 183 citations

Recombinant Retroviruses Pseudotyped With the Vesicular Stomatitis Virus G Glycoprotein Mediate Both Stable Gene Transfer and Pseudotransduction in Human Peripheral Blood Lymphocytes

1997 • 169 citations

Lentiviral Vectors--the Promise of Gene Therapy Within Reach?

1999 • 168 citations

Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure.

1994 • 162 citations

High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells.

1990 • 129 citations

Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing.

1998 • 112 citations

Do LCRs Open Chromatin Domains?

1998 • 103 citations

A second generation transgenic mouse model expressing both hemoglobin S (HbS) and HbS-Antilles results in increased phenotypic severity

1995 • 94 citations

The Human β Globin Locus Introduced by YAC Transfer Exhibits a Specific and Reproducible Pattern of Developmental Regulation in Transgenic Mice

1997 • 82 citations

Long-Term Transfer and Expression of the Human β-Globin Gene in a Mouse Transplant Model

1997 • 61 citations

Bone Marrow Transplantation for Homozygous β‐Thalassemia: The Memorial Sloan‐Kettering Cancer Center Experience

1998 • 36 citations

Thalassemia Syndromes

2014 • 28 citations

GENETIC TREATMENT OF THE HAEMOGLOINOPATHIES: RECOMBINATIONS AND NEW COMBINATIONS

1997 • 23 citations

The dynamics of globin gene expression and gene therapy vectors.

1998 • 20 citations

Synthesis of Haemoglobins specified by Allelic Genes in Human Heterozygotes

1971 • 9 citations