4.7 Article

Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration

Related references

Note: Only part of the references are listed.
Article Multidisciplinary Sciences

Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

Giulia Pavani et al.

NATURE COMMUNICATIONS (2020)

Review Biochemistry & Molecular Biology

Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

Hongyi Li et al.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2020)

Article Biochemistry & Molecular Biology

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

Christopher E. Nelson et al.

NATURE MEDICINE (2019)

Article Multidisciplinary Sciences

Gene correction for SCID-X1 in long-term hematopoietic stem cells

Mara Pavel-Dinu et al.

NATURE COMMUNICATIONS (2019)

Article Multidisciplinary Sciences

High levels of AAV vector integration into CRISPR-induced DNA breaks

Killian S. Hanlon et al.

NATURE COMMUNICATIONS (2019)

Article Multidisciplinary Sciences

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

Natalia Gomez-Ospina et al.

NATURE COMMUNICATIONS (2019)

Review Genetics & Heredity

In vivo genome editing via the HITI method as a tool for gene therapy

Keiichiro Suzuki et al.

JOURNAL OF HUMAN GENETICS (2018)

Article Biochemical Research Methods

CRISPR/Cas9 genome editing in human hematopoietic stem cells

Rasmus O. Bak et al.

NATURE PROTOCOLS (2018)

Article Hematology

Cell cycle regulation of hematopoietic stem or progenitor cells

Sha Hao et al.

INTERNATIONAL JOURNAL OF HEMATOLOGY (2016)

Article Multidisciplinary Sciences

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

Keiichiro Suzuki et al.

NATURE (2016)

Article Multidisciplinary Sciences

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Daniel P. Dever et al.

NATURE (2016)

Article Biotechnology & Applied Microbiology

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

Suk See De Ravin et al.

NATURE BIOTECHNOLOGY (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 Biotechnology & Applied Microbiology

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

Jianbin Wang et al.

NATURE BIOTECHNOLOGY (2015)

Article Multidisciplinary Sciences

Targeted genome editing in human repopulating haematopoietic stem cells

Pietro Genovese et al.

NATURE (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 Biochemical Research Methods

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer

Marco Ranzani et al.

NATURE METHODS (2013)

Article Multidisciplinary Sciences

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Alessandra Biffi et al.

SCIENCE (2013)

Article Multidisciplinary Sciences

RNA-Guided Human Genome Engineering via Cas9

Prashant Mali et al.

SCIENCE (2013)

Article Multidisciplinary Sciences

Multiplex Genome Engineering Using CRISPR/Cas Systems

Le Cong et al.

SCIENCE (2013)

Review Biotechnology & Applied Microbiology

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

Thomas Gaj et al.

TRENDS IN BIOTECHNOLOGY (2013)

Article Hematology

Hematologically important mutations: Leukocyte adhesion deficiency (first update)

Edith van de Vijver et al.

BLOOD CELLS MOLECULES AND DISEASES (2012)

Article Biochemistry & Molecular Biology

VarScan 2: Somatic mutation and copy number alteration discovery in cancer by exome sequencing

Daniel C. Koboldt et al.

GENOME RESEARCH (2012)

Article Biochemical Research Methods

FLASH: fast length adjustment of short reads to improve genome assemblies

Tanja Magoc et al.

BIOINFORMATICS (2011)

Article Cell & Tissue Engineering

Hematopoietic Stem Cell Quiescence Promotes Error-Prone DNA Repair and Mutagenesis

Mary Mohrin et al.

CELL STEM CELL (2010)

Article Biochemical Research Methods

Fast and accurate short read alignment with Burrows-Wheeler transform

Heng Li et al.

BIOINFORMATICS (2009)

Article Medicine, General & Internal

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.

Alessandro Aiuti et al.

NEW ENGLAND JOURNAL OF MEDICINE (2009)

Article Biochemistry & Molecular Biology

Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

Thomas R. Bauer et al.

NATURE MEDICINE (2008)

Article Genetics & Heredity

Adeno-associated virus vectors integrate at chromosome breakage sites

DG Miller et al.

NATURE GENETICS (2004)

Review Allergy

Gene therapy of X-linked severe combined immunodeficiency

Marina Cavazzana-Calvo et al.

CURRENT OPINION IN ALLERGY AND CLINICAL IMMUNOLOGY (2002)

Article Biotechnology & Applied Microbiology

Insect cells as a factory to produce adeno-associated virus type 2 vectors

M Urabe et al.

HUMAN GENE THERAPY (2002)

Article Hematology

Gene therapy of X-linked severe combined immunodeficiency

S Hacein-Bey-Abina et al.

INTERNATIONAL JOURNAL OF HEMATOLOGY (2002)

Article Biochemistry & Molecular Biology

Sister chromatid gene conversion is a prominent double-strand break repair pathway in mammalian cells

RD Johnson et al.

EMBO JOURNAL (2000)