Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia

Investigation of product derived lymphoma following infusion of piggyBac modified CD19 chimeric antigen receptor T-cells

(2021) Kenneth Paul Micklethwaite et al.   BLOOD

Development of CAR T-cell lymphoma in two of ten patients effectively treated with piggyBac modified CD19 CAR T-cells

(2021) David C Bishop et al.   BLOOD

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

Risk factors and outcomes in children with high-risk B-cell precursor and T-cell relapsed acute lymphoblastic leukaemia: combined analysis of ALLR3 and ALL-REZ BFM 2002 clinical trials

(2021) Cornelia Eckert et al.   EUROPEAN JOURNAL OF CANCER

Base-edited CAR T cells for combinational therapy against T cell malignancies

(2021) Christos Georgiadis et al.   LEUKEMIA

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Management of Immune-Related Adverse Events in Patients Treated With Chimeric Antigen Receptor T-Cell Therapy: ASCO Guideline

(2021) Bianca D. Santomasso et al.   JOURNAL OF CLINICAL ONCOLOGY

Off-the-shelf, gene-edited CAR-T cells forge ahead, despite safety scare

(2021) Cormac Sheridan   NATURE BIOTECHNOLOGY

CRISPR treatment inserted directly into the body for first time

(2020) Heidi Ledford   NATURE

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Exploring the Dilemma of Allogeneic Hematopoietic Cell Transplantation after Chimeric Antigen Receptor T Cell Therapy: To Transplant or Not?

(2020) Stella Bouziana et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

‘Mini’ U6 Pol III promoter exhibits nucleosome redundancy and supports multiplexed coupling of CRISPR/Cas9 effects

(2020) Roland Preece et al.   GENE THERAPY

Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer

(2020) You Lu et al.   NATURE MEDICINE

CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies

(2020) Valentín Ortíz-Maldonado et al.   MOLECULAR THERAPY

Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies

(2020) Reuben Benjamin et al.   LANCET

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

The response to lymphodepletion impacts PFS in aggressive non-Hodgkin lymphoma patients treated with CD19 CAR-T cells

(2019) Alexandre V. Hirayama et al.   BLOOD

Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases

(2019) Daesik Kim et al.   Annual Review of Biochemistry

Population Pharmacokinetics of Alemtuzumab (Campath) in Pediatric Hematopoietic Cell Transplantation: Towards Individualized Dosing to Improve Outcome

(2019) Rick Admiraal et al.   CLINICAL PHARMACOKINETICS

Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial

(2019) Wei-Li Di et al.   HUMAN GENE THERAPY

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

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

Outcome in Children With Standard-Risk B-Cell Acute Lymphoblastic Leukemia: Results of Children’s Oncology Group Trial AALL0331

(2019) Kelly W. Maloney et al.   JOURNAL OF CLINICAL ONCOLOGY

Long Terminal Repeat CRISPR-CAR-Coupled “Universal” T Cells Mediate Potent Anti-leukemic Effects

(2018) Christos Georgiadis et al.   MOLECULAR THERAPY

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

(2018) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

Locally produced CD19 CAR T cells leading to clinical remissions in medullary and extramedullary relapsed acute lymphoblastic leukemia

(2018) Elad Jacoby et al.   AMERICAN JOURNAL OF HEMATOLOGY

Disruption of TET2 promotes the therapeutic efficacy of CD19-targeted T cells

(2018) Joseph A. Fraietta et al.   NATURE

Induction of resistance to chimeric antigen receptor T cell therapy by transduction of a single leukemic B cell

(2018) Marco Ruella et al.   NATURE MEDICINE

FDA Approval of Tisagenlecleucel

(2017) Peter B. Bach et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

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

(2017) Waseem Qasim et al.   Science Translational Medicine

Automated manufacturing of chimeric antigen receptor T cells for adoptive immunotherapy using CliniMACS Prodigy

(2016) Ulrike Mock et al.   CYTOTHERAPY

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Augmented post-remission therapy for a minimal residual disease-defined high-risk subgroup of children and young people with clinical standard-risk and intermediate-risk acute lymphoblastic leukaemia (UKALL 2003): a randomised controlled trial

(2014) Ajay Vora et al.   LANCET ONCOLOGY

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Impact of viral reactivations in the era of pre-emptive antiviral drug therapy following allogeneic haematopoietic SCT in paediatric recipients

(2012) P Hiwarkar et al.   BONE MARROW TRANSPLANTATION

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

(2012) M. Jinek et al.   SCIENCE