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

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

Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease

(2016) M. D. Hoban et al.   BLOOD

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

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

(2015) M. D. Hoban et al.   BLOOD

Pomalidomide reverses  -globin silencing through the transcriptional reprogramming of adult hematopoietic progenitors

(2015) B. M. Dulmovits et al.   BLOOD

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

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

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

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

Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory

(2015) Giacomo Oliveira et al.   Science Translational Medicine

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

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

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

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal

(2014) I. Fares et al.   SCIENCE

Quantifying Genome-Editing Outcomes at Endogenous Loci with SMRT Sequencing

(2014) Ayal Hendel et al.   Cell Reports

Engraftment and Lineage Potential of Adult Hematopoietic Stem and Progenitor Cells Is Compromised Following Short-Term Culture in the Presence of an Aryl Hydrocarbon Receptor Antagonist

(2014) Angel Gu et al.   Human Gene Therapy Methods

Isolation and functional characterization of human erythroblasts at distinct stages: implications for understanding of normal and disordered erythropoiesis in vivo

(2013) J. Hu et al.   BLOOD

Gene therapy for PIDs: Progress, pitfalls and prospects

(2013) Sayandip Mukherjee et al.   GENE

β-globin gene transfer to human bone marrow for sickle cell disease

(2013) Zulema Romero et al.   JOURNAL OF CLINICAL INVESTIGATION

Nuclease-mediated gene editing by homologous recombination of the human globin locus

(2013) Richard A. Voit et al.   NUCLEIC ACIDS RESEARCH

CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity

(2013) Thomas J. Cradick et al.   NUCLEIC ACIDS RESEARCH

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

Collaboration and competition between DNA double-strand break repair pathways

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

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Hematopoietic stem cell: self-renewal versus differentiation

(2010) Jun Seita et al.   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I–II study

(2009) Fabio Ciceri et al.   LANCET ONCOLOGY