相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
Farid Boulad et al.
NATURE MEDICINE (2022)
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease
Sunita Goyal et al.
NEW ENGLAND JOURNAL OF MEDICINE (2022)
Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for b-hemoglobinopathies
Claire M. Drysdale et al.
CELL STEM CELL (2021)
Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither
Richard J. Jones et al.
BLOOD (2021)
Chromatin Conformation in Development and Disease
Ilias Boltsis et al.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY (2021)
Innovative Therapies for Hemoglobin Disorders
Karine Sii-Felice et al.
BIODRUGS (2020)
GATA-1-dependent histone H3K27 acetylation mediates erythroid cell-specific chromatin interaction between CTCF sites
Yea Woon Kim et al.
FASEB JOURNAL (2020)
HIV proviral DNA integration can drive T cell growth ex vivo
John K. Yoon et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)
Gene Therapy and Genome Editing
Farid Boulad et al.
HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA (2018)
Enhancer hubs and loop collisions identified from single-allele topologies
Amin Allahyar et al.
NATURE GENETICS (2018)
Comparative analysis of three-dimensional chromosomal architecture identifies a novel fetal hemoglobin regulatory element
Peng Huang et al.
GENES & DEVELOPMENT (2017)
Insulator dysfunction and oncogene activation in IDH mutant gliomas
William A. Flavahan et al.
NATURE (2016)
Lineage-specific and single-cell chromatin accessibility charts human hematopoiesis and leukemia evolution
M. Ryan Corces et al.
NATURE GENETICS (2016)
Disruptions of Topological Chromatin Domains Cause Pathogenic Rewiring of Gene-Enhancer Interactions
Dario G. Lupianez et al.
CELL (2015)
Genomic discovery of potent chromatin insulators for human gene therapy
Mingdong Liu et al.
NATURE BIOTECHNOLOGY (2015)
Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation
Farid Boulad et al.
BLOOD (2014)
Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
Olivier Negre et al.
CURRENT GENE THERAPY (2014)
β-globin gene transfer to human bone marrow for sickle cell disease
Zulema Romero et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
Topological domains in mammalian genomes identified by analysis of chromatin interactions
Jesse R. Dixon et al.
NATURE (2012)
3′UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice
Kazuhiko Ikeda et al.
BLOOD (2011)
The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors
David W. Emery
HUMAN GENE THERAPY (2011)
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo et al.
NATURE (2010)
Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design
Giulietta Maruggi et al.
MOLECULAR THERAPY (2009)
Elucidation of the phenotypic, functional, and molecular topography of a myeloerythroid progenitor cell hierarchy
Cornelis J. H. Pronk et al.
CELL STEM CELL (2007)
Lineage-specific transcription factors in multipotent hematopoietic progenitors - A little bit goes a long way
Stefania Bottardi et al.
CELL CYCLE (2007)
Replication and transcription: Shaping the landscape of the genome
L Chakalova et al.
NATURE REVIEWS GENETICS (2005)
Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia
H Hanawa et al.
BLOOD (2004)
The active spatial organization of the β-globin locus requires the transcription factor EKLF
R Drissen et al.
GENES & DEVELOPMENT (2004)
Correction of a mouse model of sickle cell disease:: lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells
DN Levasseur et al.
BLOOD (2003)
Developmental stage-specific epigenetic control of human β-globin gene expression is potentiated in hematopoietic progenitor cells prior to their transcriptional activation
S Bottardi et al.
BLOOD (2003)
The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number
DA Persons et al.
BLOOD (2003)
HIV-1 integration in the human genome favors active genes and local hotspots
ARW Schröder et al.
CELL (2002)
Correction of sickle cell disease in transgenic mouse models by gene therapy
R Pawliuk et al.
SCIENCE (2001)
Distinct mechanisms control RNA polymerase II recruitment to a tissue-specific locus control region and a downstream promoter
KD Johnson et al.
MOLECULAR CELL (2001)
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
C May et al.
NATURE (2000)
The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites
S Rivella et al.
JOURNAL OF VIROLOGY (2000)
Hemopoietic lineage commitment decisions:: in vivo evidence from a transgenic mouse model harboring μLCR-βpro-LacZ as a transgene
T Papayannopoulou et al.
BLOOD (2000)