CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

Pivotal Safety and Efficacy Results from Transcend NHL 001, a Multicenter Phase 1 Study of Lisocabtagene Maraleucel (liso-cel) in Relapsed/Refractory (R/R) Large B Cell Lymphomas

(2020) Jeremy S. Abramson et al.   BLOOD

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Effective multi-lineage engraftment in a mouse model of Fanconi anemia using non-genotoxic antibody-based conditioning

(2020) Meera A. Srikanthan et al.   Molecular Therapy-Methods & Clinical Development

A20 and RBX1 regulate brentuximab vedotin sensitivity in Hodgkin Lymphoma models

(2020) Wei Wei et al.   CLINICAL CANCER RESEARCH

Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation

(2020) Volker Wiebking et al.   HAEMATOLOGICA

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants

(2020) Russell T. Walton et al.   SCIENCE

DOT1L inhibition is lethal for multiple myeloma due to perturbation of the endoplasmic reticulum stress pathway

(2020) Caroline Dafflon et al.   Oncotarget

CRISPR/Cas9-Mediated Foxp1 Silencing Restores Immune Surveillance in an Immunocompetent A20 Lymphoma Model

(2020) Suet Ling Felce et al.   Frontiers in Oncology

Anti-human CD117 antibody-mediated bone marrow niche clearance in non-human primates and humanized NSG mice

(2019) Hye-Sook Kwon et al.   BLOOD

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

(2019) Christopher T. Lux et al.   Molecular Therapy-Methods & Clinical Development

Hematopoietic chimerism and donor-specific skin allograft tolerance after non-genotoxic CD117 antibody-drug-conjugate conditioning in MHC-mismatched allotransplantation

(2019) Zhanzhuo Li et al.   Nature Communications

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Engineered CRISPR–Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing

(2019) Benjamin P. Kleinstiver et al.   NATURE BIOTECHNOLOGY

HIV-1 remission following CCR5Δ32/Δ32 haematopoietic stem-cell transplantation

(2019) Ravindra K. Gupta et al.   NATURE

CRISPR to fix bad blood: a new tool in basic and clinical hematology

(2019) Elisa González-Romero et al.   HAEMATOLOGICA

Editing the Sickle Cell Disease Mutation in Primary Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

(2019) Zulema Romero et al.   MOLECULAR THERAPY

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

(2019) Reza Shahbazi et al.   NATURE MATERIALS

Molecular scissors cut in on stem cells

(2019) Anna Azvolinsky   NATURE MEDICINE

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

(2019) Ewelina Mamcarz et al.   NEW ENGLAND JOURNAL OF MEDICINE

A novel model of controlling PD-L1 expression in ALK+ anaplastic large cell lymphoma revealed by CRISPR screening

(2019) Jing-Ping Zhang et al.   BLOOD

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

(2019) Olivier Humbert et al.   Science Translational Medicine

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

(2019) Mara Pavel-Dinu et al.   Nature Communications

Sequential LASER ART and CRISPR Treatments Eliminate HIV-1 in a Subset of Infected Humanized Mice

(2019) Prasanta K. Dash et al.   Nature Communications

NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia

(2019) Francisco José Román-Rodríguez et al.   Cell Stem Cell

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

(2019) Paula Río et al.   NATURE MEDICINE

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

(2019) Lei Xu et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

(2019) Sowmya Pattabhi et al.   Molecular Therapy-Nucleic Acids

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Potent efficacy of MCL-1 inhibitor-based therapies in preclinical models of mantle cell lymphoma

(2019) Michael A. Dengler et al.   ONCOGENE

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

(2019) Natalia Gomez-Ospina et al.   Nature Communications

Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors

(2019) Beau R. Webber et al.   Nature Communications

Molecular basis for the PAM expansion and fidelity enhancement of an evolved Cas9 nuclease

(2019) Weizhong Chen et al.   PLOS BIOLOGY

Endogenous CRISPR-Cas System-Based Genome Editing and Antimicrobials: Review and Prospects

(2019) Yingjun Li et al.   Frontiers in Microbiology

Advances in genome editing through control of DNA repair pathways

(2019) Charles D. Yeh et al.   NATURE CELL BIOLOGY

Gene therapy and gene editing strategies for hemoglobinopathies

(2018) Maria Rosa Lidonnici et al.   BLOOD CELLS MOLECULES AND DISEASES

Counterpoint: The Potential Harms of Human Gene Editing Using CRISPR-Cas9

(2018) Françoise Baylis   CLINICAL CHEMISTRY

Gene therapy for sickle cell disease: An update

(2018) Selami Demirci et al.   CYTOTHERAPY

Coexpression profile of leukemic stem cell markers for combinatorial targeted therapy in AML

(2018) S. Haubner et al.   LEUKEMIA

Myeloid Conditioning with c-kit-Targeted CAR-T Cells Enables Donor Stem Cell Engraftment

(2018) Yasuyuki Arai et al.   MOLECULAR THERAPY

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34 + Hematopoietic Stem and Progenitor Cells

(2018) M. Kyle Cromer et al.   MOLECULAR THERAPY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

CRISPR/Cas9 genome editing in human hematopoietic stem cells

(2018) Rasmus O Bak et al.   Nature Protocols

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Multiplexed and portable nucleic acid detection platform with Cas13, Cas12a, and Csm6

(2018) Jonathan S. Gootenberg et al.   SCIENCE

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

(2018) Jayesh A. Kulkarni et al.   Nucleic Acid Therapeutics

Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy

(2018) Christopher W. Peterson et al.   PLoS Pathogens

Efficient Enrichment of Gene-Modified Primary T Cells via CCR5-Targeted Integration of Mutant Dihydrofolate Reductase

(2018) Biswajit Paul et al.   Molecular Therapy-Methods & Clinical Development

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri et al.   Molecular Therapy-Methods & Clinical Development

HDAd5/35 ++ Adenovirus Vector Expressing Anti-CRISPR Peptides Decreases CRISPR/Cas9 Toxicity in Human Hematopoietic Stem Cells

(2018) Chang Li et al.   Molecular Therapy-Methods & Clinical Development

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

The STING-STAT6 pathway drives Cas9-induced host response in human monocytes

(2018) Rui Kang et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

(2018) Hongjie Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Engineering resistance to CD33-targeted immunotherapy in normal hematopoiesis by CRISPR/Cas9-deletion of CD33 exon 2

(2018) Olivier Humbert et al.   LEUKEMIA

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

Genome-edited baby claim provokes international outcry

(2018) David Cyranoski et al.   NATURE

Sangamo's landmark genome editing trial gets mixed reception

(2018) Cormac Sheridan   NATURE BIOTECHNOLOGY

Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

(2018) Rasmus O Bak et al.   eLife

Allogeneic CAR-T Cells: More than Ease of Access?

(2018) Charlotte Graham et al.   Cells

Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency

(2017) Kit L. Shaw et al.   JOURNAL OF CLINICAL INVESTIGATION

Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers

(2017) So Gun Hong et al.   MOLECULAR THERAPY

CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

(2017) Lei Xu et al.   MOLECULAR THERAPY

Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection

(2017) Justin Eyquem et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells

(2017) Germán G Gornalusse et al.   NATURE BIOTECHNOLOGY

Refining strategies to translate genome editing to the clinic

(2017) Tatjana I Cornu et al.   NATURE MEDICINE

Unexpected mutations after CRISPR–Cas9 editing in vivo

(2017) Kellie A Schaefer et al.   NATURE METHODS

Marker-free coselection for CRISPR-driven genome editing in human cells

(2017) Daniel Agudelo et al.   NATURE METHODS

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nucleic acid detection with CRISPR-Cas13a/C2c2

(2017) Jonathan S. Gootenberg et al.   SCIENCE

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

(2017) Stefan Radtke et al.   Science Translational Medicine

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

(2017) Waseem Qasim et al.   Science Translational Medicine

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

(2017) Kai-Hsin Chang et al.   Molecular Therapy-Methods & Clinical Development

Disabling Cas9 by an anti-CRISPR DNA mimic

(2017) Jiyung Shin et al.   Science Advances

Genome Editing: A New Approach to Human Therapeutics

(2016) Matthew Porteus   Annual Review of Pharmacology and Toxicology

Multiplex Genome Editing to Generate Universal CAR T Cells Resistant to PD1 Inhibition

(2016) Jiangtao Ren et al.   CLINICAL CANCER RESEARCH

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

(2016) Daniel P. Dever et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

(2016) Rahul Palchaudhuri et al.   NATURE BIOTECHNOLOGY

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

(2016) Elizabeth A Traxler et al.   NATURE MEDICINE

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

(2016) Lin Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

(2015) Laurent Poirot et al.   CANCER RESEARCH

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Nonirradiated NOD,B6.SCID Il2rγ−/− KitW41/W41 (NBSGW) Mice Support Multilineage Engraftment of Human Hematopoietic Cells

(2015) Brian E. McIntosh et al.   Stem Cell Reports

Peripheral Blood Progenitor Cell Mobilization for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation

(2014) Hien K. Duong et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

(2014) Luke A. Gilbert et al.   CELL

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9

(2014) Pankaj K. Mandal et al.   Cell Stem Cell

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

Microhomology-based choice of Cas9 nuclease target sites

(2014) Sangsu Bae et al.   NATURE METHODS

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene-modified hematopoietic stem cells for cancer immunotherapy

(2014) Sarah Larson et al.   Human Vaccines & Immunotherapeutics

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

(2013) Sandrine Boissel et al.   NUCLEIC ACIDS RESEARCH

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Chemoselection of Allogeneic HSC After Murine Neonatal Transplantation Without Myeloablation or Post-transplant Immunosuppression

(2012) Rustom Falahati et al.   MOLECULAR THERAPY

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2012) M. Jinek et al.   SCIENCE

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates

(2010) Brian C. Beard et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

(2010) Ting Li et al.   NUCLEIC ACIDS RESEARCH

Granulocyte colony-stimulating factor (G-CSF) administration in individuals with sickle cell disease: time for a moratorium?

(2009) Courtney D. Fitzhugh et al.   CYTOTHERAPY

Long-Term Control of HIV byCCR5Delta32/Delta32 Stem-Cell Transplantation

(2009) Gero Hütter et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY