Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T‐cells for the treatment of Hyper IgM1

Cell cycle arrest and p53 prevent ON-target megabase-scale rearrangements induced by CRISPR-Cas9

(2023) G. Cullot et al.   Nature Communications

Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo

(2023) Claudia Asperti et al.   Molecular Therapy-Methods & Clinical Development

Optical genome mapping for structural variation analysis in hematologic malignancies

(2022) Adam C. Smith et al.   AMERICAN JOURNAL OF HEMATOLOGY

Frequent aneuploidy in primary human T cells after CRISPR–Cas9 cleavage

(2022) Alessio David Nahmad et al.   NATURE BIOTECHNOLOGY

CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia

(2022) Bin Fu et al.   NATURE MEDICINE

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

(2022) Samuele Ferrari et al.   Cell Stem Cell

Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing

(2022) So Hyun Park et al.   Science Advances

Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells

(2022) Barbra J. Sasu et al.   MOLECULAR THERAPY

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome

(2021) Valentina Vavassori et al.   EMBO Molecular Medicine

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

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

BAR-Seq clonal tracking of gene-edited cells

(2021) Samuele Ferrari et al.   Nature Protocols

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells

(2021) J. Boutin et al.   Nature Communications

Human Inborn Errors of Immunity: 2019 Update on the Classification from the International Union of Immunological Societies Expert Committee

(2020) Stuart G. Tangye et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

(2020) Samuele Ferrari et al.   NATURE BIOTECHNOLOGY

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

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

Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study

(2019) Francesca Ferrua et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

A Survey of Validation Strategies for CRISPR-Cas9 Editing

(2018) Monica F. Sentmanat et al.   Scientific Reports

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

(2018) Caroline Y. Kuo et al.   Cell Reports

Large deletions induced by Cas9 cleavage

(2018) Fatwa Adikusuma et al.   NATURE

The hyper IgM syndromes: Epidemiology, pathogenesis, clinical manifestations, diagnosis and management

(2018) Reza Yazdani et al.   CLINICAL IMMUNOLOGY

European guidelines for constitutional cytogenomic analysis

(2018) Marisa Silva et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

(2017) M. Teresa de la Morena et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

(2017) Giulia Schiroli et al.   Science Translational Medicine

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

(2016) Nicholas Hubbard et al.   BLOOD