(Q57008552)

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

21 January 2018

1 reference

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

21 January 2018

1 reference

The vagaries of variegating transgenes

21 January 2018

1 reference

Locus control regions: overcoming heterochromatin-induced gene inactivation in mammals

21 January 2018

1 reference

Gene silencing as a threat to the success of gene therapy

21 January 2018

1 reference

Silencing and variegation of gammaretrovirus and lentivirus vectors

21 January 2018

1 reference

Position-effect variegation and the new biology of heterochromatin

21 January 2018

1 reference

Heterochromatin and gene expression in Drosophila

21 January 2018

1 reference

Mode of proviral activation of a putative mammary oncogene (int-1) on mouse chromosome 15

21 January 2018

1 reference

HIV-1 integration in the human genome favors active genes and local hotspots

21 January 2018

1 reference

Transcription start regions in the human genome are favored targets for MLV integration

21 January 2018

1 reference

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

21 January 2018

1 reference

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

21 January 2018

1 reference

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

21 January 2018

1 reference

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

21 January 2018

1 reference

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

21 January 2018

1 reference

Dose finding with retroviral vectors: correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population

21 January 2018

1 reference

Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis.

21 January 2018

1 reference

The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors

21 January 2018

1 reference

LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin

21 January 2018

1 reference

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

21 January 2018

1 reference

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

21 January 2018

1 reference

The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors

21 January 2018

1 reference

Solving the problem of γ-retroviral vectors containing long terminal repeats

21 January 2018

1 reference

Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century

21 January 2018

1 reference

Mitotic homologous recombination maintains genomic stability and suppresses tumorigenesis

21 January 2018

1 reference

Genetic manipulation of genomes with rare-cutting endonucleases

21 January 2018

1 reference

Gene targeting using zinc finger nucleases

21 January 2018

1 reference

Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy

21 January 2018

1 reference

TALEs of genome targeting

21 January 2018

1 reference

RTCGD: retroviral tagged cancer gene database

21 January 2018

1 reference

Genome-based identification of cancer genes by proviral tagging in mouse retrovirus-induced T-cell lymphomas

21 January 2018

1 reference

Occurrence of leukaemia following gene therapy of X-linked SCID.

21 January 2018

1 reference

Efficacy of gene therapy for X-linked severe combined immunodeficiency

21 January 2018

1 reference

CancerGenes: a gene selection resource for cancer genome projects

21 January 2018

1 reference

Atlas of genetics and cytogenetics in oncology and haematology, an interactive database

21 January 2018

1 reference

The consensus coding sequences of human breast and colorectal cancers

21 January 2018

1 reference

A census of human cancer genes

21 January 2018

1 reference

Integrated genomic analyses of ovarian carcinoma

21 January 2018

1 reference

Site-specific integration and tailoring of cassette design for sustainable gene transfer.

21 January 2018

1 reference

Initial sequencing and analysis of the human genome

21 January 2018

1 reference

Interspersed repeats and other mementos of transposable elements in mammalian genomes

21 January 2018

1 reference

Integration target site selection by a resurrected human endogenous retrovirus

21 January 2018

1 reference

Integration targeting by avian sarcoma-leukosis virus and human immunodeficiency virus in the chicken genome

21 January 2018

1 reference

Adeno-associated virus: a key to the human genome?

21 January 2018

1 reference

Characterization of adeno-associated virus genomes isolated from human tissues

21 January 2018

1 reference

Adeno-associated viruses undergo substantial evolution in primates during natural infections

21 January 2018

1 reference

Increased complexity of wild-type adeno-associated virus-chromosomal junctions as determined by analysis of unselected cellular genomes

21 January 2018

1 reference

Integration preferences of wildtype AAV-2 for consensus rep-binding sites at numerous loci in the human genome

21 January 2018

1 reference

Integration of adeno-associated virus (AAV) and recombinant AAV vectors

21 January 2018

1 reference

Evidence of chromosomal integration of AAV DNA in human testis tissue.

21 January 2018

1 reference

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

21 January 2018

1 reference

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting

21 January 2018

1 reference

Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors

21 January 2018

1 reference

Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration

21 January 2018

1 reference

Human cardiovascular progenitor cells develop from a KDR+ embryonic-stem-cell-derived population

21 January 2018

1 reference

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

21 January 2018

1 reference

Site-specific integration of adeno-associated virus involves partial duplication of the target locus.

21 January 2018

1 reference

Characterization of the mouse adeno-associated virus AAVS1 ortholog.

21 January 2018

1 reference

Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA.

21 January 2018

1 reference

Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection

21 January 2018

1 reference

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

21 January 2018

1 reference

CCR5: no longer a "good for nothing" gene--chemokine control of West Nile virus infection

21 January 2018

1 reference

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

1 reference

Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells

21 January 2018

1 reference

Identification and targeting of the ROSA26 locus in human embryonic stem cells.

21 January 2018

1 reference

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

21 January 2018

1 reference

21 January 2018

Locus control regions

1 reference

Ultraconserved Elements in the Human Genome

21 January 2018

1 reference

Transcriptional control thrown for a loop

21 January 2018

1 reference

Origin of phenotypes: genes and transcripts

21 January 2018

1 reference

Methods for integration site distribution analyses in animal cell genomes

21 January 2018

1 reference

Comprehensive genomic access to vector integration in clinical gene therapy

21 January 2018

1 reference

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

21 January 2018

1 reference

High-throughput transgenesis in Xenopus using I-SceI meganuclease

21 January 2018

1 reference

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

21 January 2018

1 reference

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

21 January 2018

1 reference

Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination

21 January 2018

1 reference

12 December 2020

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

1 reference

12 December 2020

Hematopoietic stem cell engineering at a crossroads

1 reference

12 December 2020

Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model

1 reference

12 December 2020

Reconstructing blood from induced pluripotent stem cells

1 reference

12 December 2020

Cellular localization of the chemokine receptor CCR5. Correlation to cellular targets of HIV-1 infection.

1 reference

12 December 2020