High-yield genome engineering in primary cells using a hybrid ssDNA repair template and small-molecule cocktails

HLA-independent T cell receptors for targeting tumors with low antigen density

(2022) Jorge Mansilla-Soto et al.   NATURE MEDICINE

Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells

(2022) Jonas Kath et al.   Molecular Therapy-Methods & Clinical Development

Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma

(2021) Nikhil C. Munshi et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2021) Kenneth Paul Micklethwaite et al.   BLOOD

Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing

(2021) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Human Inborn Errors of Immunity: 2019 Update of the IUIS Phenotypical Classification

(2020) Aziz Bousfiha et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Targeting DNA-PK in cancer

(2020) Giovanna Damia   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies

(2020) Theodore L. Roth et al.   CELL

Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair

(2020) Beeke Wienert et al.   Nature Communications

Clinical, immunological, and genetic features in patients with Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-linked (IPEX) and IPEX-like Syndrome

(2020) Mahnaz Jamee et al.   Journal of Allergy and Clinical Immunology-In Practice

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

(2020) M. Goodwin et al.   Science Advances

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Rational selection of CRISPR/Cas9 guide RNAs for homology directed genome editing

(2020) Kristina J. Tatiossian et al.   MOLECULAR THERAPY

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

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

Dynamics and competition of CRISPR–Cas9 ribonucleoproteins and AAV donor-mediated NHEJ, MMEJ and HDR editing

(2020) Ya-Wen Fu et al.   NUCLEIC ACIDS RESEARCH

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

(2019) Renata M. Martin et al.   Cell Stem Cell

CD25 deficiency: A new conformational mutation prevents the receptor expression on cell surface

(2019) Marina Vignoli et al.   CLINICAL IMMUNOLOGY

A Dual Inhibitor of Cdc7/Cdk9 Potently Suppresses T Cell Activation

(2019) Elijah W. Chen et al.   Frontiers in Immunology

NextGen cell-based immunotherapies in cancer and other immune disorders

(2019) Camillia S. Azimi et al.   CURRENT OPINION IN IMMUNOLOGY

Effective and safe treatment of a novel IL2RA deficiency with rapamycin

(2019) Nannan Lai et al.   Journal of Allergy and Clinical Immunology-In Practice

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency

(2019) David N. Nguyen et al.   NATURE BIOTECHNOLOGY

Advances in genome editing through control of DNA repair pathways

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

Phenotype, penetrance, and treatment of 133 cytotoxic T-lymphocyte antigen 4–insufficient subjects

(2018) Charlotte Schwab et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Easy quantification of template-directed CRISPR/Cas9 editing

(2018) Eva K Brinkman et al.   NUCLEIC ACIDS RESEARCH

Reprogramming human T cell function and specificity with non-viral genome targeting

(2018) Theodore L. Roth et al.   NATURE

Accurate classification of BRCA1 variants with saturation genome editing

(2018) Gregory M. Findlay 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

Calibration of CAR activation potential directs alternative T cell fates and therapeutic potency

(2018) Judith Feucht et al.   NATURE MEDICINE

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

(2017) Justin Eyquem et al.   NATURE

Co-incident insertion enables high efficiency genome engineering in mouse embryonic stem cells

(2016) Brian R. Shy et al.   NUCLEIC ACIDS RESEARCH

Variation in the CTLA4 3′UTR has phenotypic consequences for autoreactive T cells and associates with genetic risk for type 1 diabetes

(2015) V M de Jong et al.   GENES AND IMMUNITY

Cas9 gRNA engineering for genome editing, activation and repression

(2015) Samira Kiani et al.   NATURE METHODS

Targeted sequencing by proximity ligation for comprehensive variant detection and local haplotyping

(2014) Paula J P de Vree et al.   NATURE BIOTECHNOLOGY

Autosomal dominant immune dysregulation syndrome in humans with CTLA4 mutations

(2014) Desirée Schubert et al.   NATURE MEDICINE

Immune dysregulation in human subjects with heterozygous germline mutations in CTLA4

(2014) H. S. Kuehn et al.   SCIENCE

Follicular bronchiolitis as phenotype associated with CD25 deficiency

(2013) L. Bezrodnik et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity

(2013) Kevin Goudy et al.   CLINICAL IMMUNOLOGY

Rigid-body Ligand Recognition Drives Cytotoxic T-lymphocyte Antigen 4 (CTLA-4) Receptor Triggering

(2010) Chao Yu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Manufacturing and characterizing AAV-based vectors for use in clinical studies

(2008) J F Wright   GENE THERAPY