CRISPR/Cas9 genome editing in human hematopoietic stem cells

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

(2017) Suk See De Ravin et al.   Science Translational Medicine

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

(2017) Rasmus O. Bak et al.   Cell Reports

Genome Editing: A New Approach to Human Therapeutics

(2016) Matthew Porteus   Annual Review of Pharmacology and Toxicology

Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome

(2016) Nicholas Hubbard et al.   BLOOD

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

(2016) Mark J Osborn et al.   MOLECULAR THERAPY

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

(2016) Suk See De Ravin et al.   NATURE BIOTECHNOLOGY

A humanized bone marrow ossicle xenotransplantation model enables improved engraftment of healthy and leukemic human hematopoietic cells

(2016) Andreas Reinisch et al.   NATURE MEDICINE

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

(2016) M. A. DeWitt et al.   Science Translational Medicine

Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene

(2016) Jennifer A. Hollywood et al.   Scientific Reports

Highly Efficient Genome Editing of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9

(2016) Michael C. Gundry et al.   Cell Reports

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

(2015) Jianbin Wang et al.   NUCLEIC ACIDS RESEARCH

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

Quantifying Genome-Editing Outcomes at Endogenous Loci with SMRT Sequencing

(2014) Ayal Hendel et al.   Cell Reports

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

An Efficient Low Cost Method for Gene Transfer to T Lymphocytes

(2013) Leonardo Chicaybam et al.   PLoS One

High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo

(2013) Liujiang Song et al.   PLoS One

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Hematopoiesis: A Human Perspective

(2012) Sergei Doulatov et al.   Cell Stem Cell

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

(2012) Christine Aurnhammer et al.   Human Gene Therapy Methods

AAV-mediated gene targeting methods for human cells

(2011) Iram F Khan et al.   Nature Protocols

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment

(2011) F. Notta et al.   SCIENCE

Engraftment of human hematopoietic stem cells is more efficient in female NOD/SCID/IL-2Rgc-null recipients

(2010) F. Notta et al.   BLOOD

Collaboration and competition between DNA double-strand break repair pathways

(2010) Elizabeth M. Kass et al.   FEBS LETTERS

Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material

(2010) Martin Lock et al.   HUMAN GENE THERAPY

In vivo evaluation of human hematopoiesis through xenotransplantation of purified hematopoietic stem cells from umbilical cord blood

(2009) Christopher Y Park et al.   Nature Protocols