4.8 Article

Integrome signatures of lentiviral gene therapy for SCID-X1 patients

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。
Article Multidisciplinary Sciences

Comprehensive analysis of single cell ATAC-seq data with SnapATAC

Rongxin Fang et al.

Summary: The paper introduces SnapATAC, a software package for analyzing single cell ATAC-seq datasets, which can dissect cellular heterogeneity and map cellular states' trajectories. The Nystrom method allows processing data from up to a million cells, and it incorporates existing tools for single cell ATAC-seq dataset analysis. SnapATAC is applied to mouse secondary motor cortex profiles and identifies candidate regulatory elements and cell-type specific transcriptional regulators.

NATURE COMMUNICATIONS (2021)

Article Multidisciplinary Sciences

Lentivirus-mediated gene therapy for Fabry disease

Aneal Khan et al.

Summary: Enzyme and chaperone therapies for Fabry disease are expensive, intrusive, and not fully efficacious. A pilot study was conducted using gene therapy with autologous, lentivirus-transduced hematopoietic cells expressing alpha-galactosidase A, showing safety and promising results in five patients.

NATURE COMMUNICATIONS (2021)

Article Medicine, Research & Experimental

CD19-targeting CAR T cell immunotherapy outcomes correlate with genomic modification by vector integration

Christopher L. Nobles et al.

JOURNAL OF CLINICAL INVESTIGATION (2020)

Article Multidisciplinary Sciences

Joint profiling of chromatin accessibility and CAR-T integration site analysis at population and single-cell levels T cells

Wenliang Wang et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)

Article Biochemistry & Molecular Biology

Large DNA Methylation Nadirs Anchor Chromatin Loops Maintaining Hematopoietic Stem Cell Identity

Xiaotian Zhang et al.

MOLECULAR CELL (2020)

Article Multidisciplinary Sciences

Expanded encyclopaedias of DNA elements in the human and mouse genomes

Jill E. Moore et al.

NATURE (2020)

Article Medicine, Research & Experimental

The Art and Science of Selecting a CD123-Specific Chimeric Antigen Receptor for Clinical Testing

Janice M. Riberdy et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)

Review Biotechnology & Applied Microbiology

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

Marina Cavazzana et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Article Medicine, General & Internal

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Ewelina Mamcarz et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Review Immunology

3D Chromosomal Landscapes in Hematopoiesis and Immunity

Andreas Kloetgen et al.

TRENDS IN IMMUNOLOGY (2019)

Article Multidisciplinary Sciences

Spatially clustered loci with multiple enhancers are frequent targets of HIV-1 integration

Bojana Lucic et al.

NATURE COMMUNICATIONS (2019)

Article Multidisciplinary Sciences

Revealing Hi-C subcompartments by imputing inter-chromosomal chromatin interactions

Kyle Xiong et al.

NATURE COMMUNICATIONS (2019)

Article Biochemistry & Molecular Biology

COSMIC: the Catalogue Of Somatic Mutations In Cancer

John G. Tate et al.

NUCLEIC ACIDS RESEARCH (2019)

Review Oncology

Clinical use of lentiviral vectors

Michael C. Milone et al.

LEUKEMIA (2018)

Article Biotechnology & Applied Microbiology

Integrating single-cell transcriptomic data across different conditions, technologies, and species

Andrew Butler et al.

NATURE BIOTECHNOLOGY (2018)

Review Medicine, General & Internal

Chimeric Antigen Receptor Therapy

Carl H. June et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Multidisciplinary Sciences

Coactivator condensation at super-enhancers links phase separation and gene control

Benjamin R. Sabari et al.

SCIENCE (2018)

Article Multidisciplinary Sciences

A pathway for mitotic chromosome formation

Johan H. Gibcus et al.

SCIENCE (2018)

Review Multidisciplinary Sciences

Gene therapy comes of age

Cynthia E. Dunbar et al.

SCIENCE (2018)

Review Medicine, Research & Experimental

Interactions between Retroviruses and the Host Cell Genome

Valentina Poletti et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)

Review Cell Biology

The nuclear pore complex: understanding its function through structural insight

Martin Beck et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)

Article Biochemical Research Methods

chromVAR : inferring transcription-factor-associated accessibility from single-cell epigenomic data

Alicia N. Schep et al.

NATURE METHODS (2017)

Article Medicine, General & Internal

Gene Therapy in a Patient with Sickle Cell Disease

Jean-Antoine Ribeil et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Multidisciplinary Sciences

Chromatin organization regulates viral egress dynamics

Vesa Aho et al.

SCIENTIFIC REPORTS (2017)

Article Medicine, Research & Experimental

INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions

Charles C. Berry et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)

Review Cell & Tissue Engineering

Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned

Richard A. Morgan et al.

CELL STEM CELL (2017)

Article Cell Biology

Getting to and through the inner nuclear membrane during herpesvirus nuclear egress

Ming F. Lye et al.

CURRENT OPINION IN CELL BIOLOGY (2017)

Article Biochemistry & Molecular Biology

dbSUPER: a database of super-enhancers in mouse and human genome

Aziz Khan et al.

NUCLEIC ACIDS RESEARCH (2016)

Review Biochemistry & Molecular Biology

The Nuclear Pore Complex as a Flexible and DynamicGate

Kevin E. Knockenhauer et al.

Review Cell Biology

Nucleoporins and chromatin metabolism

Christopher Ptak et al.

CURRENT OPINION IN CELL BIOLOGY (2016)

Article Cell Biology

TAD disruption as oncogenic driver

Anne-Laure Valton et al.

CURRENT OPINION IN GENETICS & DEVELOPMENT (2016)

Article Cell Biology

Nucleoporin-mediated regulation of cell identity genes

Arkaitz Ibarra et al.

GENES & DEVELOPMENT (2016)

Article Biotechnology & Applied Microbiology

Clonal Dynamics In Vivo of Virus Integration Sites of T Cells Expressing a Safety Switch

Edmund C. Chang et al.

MOLECULAR THERAPY (2016)

Article Cell Biology

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Suk See De Ravin et al.

SCIENCE TRANSLATIONAL MEDICINE (2016)

Article Biochemistry & Molecular Biology

Juicebox Provides a Visualization System for Hi-C Contact Maps with Unlimited Zoom

Neva C. Durand et al.

CELL SYSTEMS (2016)

Review Biochemistry & Molecular Biology

Retroviral integration: Site matters: Mechanisms and consequences of retroviral integration site selection

Jonas Demeulemeester et al.

BIOESSAYS (2015)

Article Multidisciplinary Sciences

Integrative analysis of 111 reference human epigenomes

Anshul Kundaje et al.

NATURE (2015)

Article Multidisciplinary Sciences

Nuclear architecture dictates HIV-1 integration site selection

Bruna Marini et al.

NATURE (2015)

Article Multidisciplinary Sciences

Chromatin organization at the nuclear pore favours HIV replication

Mickael Lelek et al.

NATURE COMMUNICATIONS (2015)

Review Immunology

Adoptive Immunotherapy for Cancer or Viruses

Marcela V. Maus et al.

ANNUAL REVIEW OF IMMUNOLOGY, VOL 32 (2014)

Article Biochemical Research Methods

circlize implements and enhances circular visualization in R

Zuguang Gu et al.

BIOINFORMATICS (2014)

Article Biochemical Research Methods

bwtool: a tool for bigWig files

Andy Pohl et al.

BIOINFORMATICS (2014)

Article Biochemistry & Molecular Biology

A 3D Map of the Human Genome at Kilobase Resolution Reveals Principles of Chromatin Looping

Suhas S. P. Rao et al.

Article Virology

Enhancers Are Major Targets for Murine Leukemia Virus Vector Integration

Suk See De Ravin et al.

JOURNAL OF VIROLOGY (2014)

Article Biotechnology & Applied Microbiology

Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo

Daniela Cesana et al.

MOLECULAR THERAPY (2014)

Review Genetics & Heredity

CTCF: an architectural protein bridging genome topology and function

Chin-Tong Ong et al.

NATURE REVIEWS GENETICS (2014)

Article Medicine, General & Internal

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

S. Hacein-Bey-Abina et al.

NEW ENGLAND JOURNAL OF MEDICINE (2014)

Article Cell Biology

Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity

Christian Joerg Braun et al.

SCIENCE TRANSLATIONAL MEDICINE (2014)

Article Biochemistry & Molecular Biology

Chromatin Position Effects Assayed by Thousands of Reporters Integrated in Parallel

Waseem Akhtar et al.

Article Biochemistry & Molecular Biology

Super-Enhancers in the Control of Cell Identity and Disease

Denes Hnisz et al.

Article Multidisciplinary Sciences

Organization of the Mitotic Chromosome

Natalia Naumova et al.

SCIENCE (2013)

Article Multidisciplinary Sciences

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Alessandro Aiuti et al.

SCIENCE (2013)

Article Multidisciplinary Sciences

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Alessandra Biffi et al.

SCIENCE (2013)

Letter Biochemical Research Methods

ChromHMM: automating chromatin-state discovery and characterization

Jason Ernst et al.

NATURE METHODS (2012)

Article Biochemical Research Methods

BEDTools: a flexible suite of utilities for comparing genomic features

Aaron R. Quinlan et al.

BIOINFORMATICS (2010)

Article Multidisciplinary Sciences

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

Marina Cavazzana-Calvo et al.

NATURE (2010)

Article Medicine, General & Internal

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.

Alessandro Aiuti et al.

NEW ENGLAND JOURNAL OF MEDICINE (2009)

Article Multidisciplinary Sciences

Comprehensive Mapping of Long-Range Interactions Reveals Folding Principles of the Human Genome

Erez Lieberman-Aiden et al.

SCIENCE (2009)

Article Medicine, Research & Experimental

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Salima Hacein-Bey-Abina et al.

JOURNAL OF CLINICAL INVESTIGATION (2008)

Article Biotechnology & Applied Microbiology

Remodelling of the nuclear lamina during human cytomegalovirus infection: role of the viral proteins pUL50 and pUL53

Daria Camozzi et al.

JOURNAL OF GENERAL VIROLOGY (2008)

Article Biotechnology & Applied Microbiology

Model-based Analysis of ChIP-Seq (MACS)

Yong Zhang et al.

GENOME BIOLOGY (2008)

Article Biochemistry & Molecular Biology

A role for LEDGF/p75 in targeting HIV DNA integration

A Ciuffi et al.

NATURE MEDICINE (2005)

Article Biochemistry & Molecular Biology

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

RS Mitchell et al.

PLOS BIOLOGY (2004)

Article Multidisciplinary Sciences

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

S Hacein-Bey-Abina et al.

SCIENCE (2003)

Article Biochemistry & Molecular Biology

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

ARW Schröder et al.