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

Lipid Nanoparticles Allow Efficient and Harmless Ex Vivo Gene Editing of Human Hematopoietic Cells

(2023) Valentina Vavassori et al.   BLOOD

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

(2023) Samuele Ferrari et al.   Cell Stem Cell

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

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

TCR-engineered T cells targeting E7 for patients with metastatic HPV-associated epithelial cancers

(2021) Nisha B. Nagarsheth et al.   NATURE 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

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

(2019) Juliane W. Schott et al.   Molecular Therapy-Methods & Clinical Development

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

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

Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

(2018) Carolina Petrillo et al.   Cell Stem Cell

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

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

(2016) Nicholas Hubbard et al.   BLOOD

Costimulatory and Coinhibitory Receptor Pathways in Infectious Disease

(2016) John Attanasio et al.   IMMUNITY

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

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

The Hyper IgM Syndromes

(2013) Nashmia Qamar et al.   CLINICAL REVIEWS IN ALLERGY & IMMUNOLOGY

SAMHD1 restricts the replication of human immunodeficiency virus type 1 by depleting the intracellular pool of deoxynucleoside triphosphates

(2012) Hichem Lahouassa et al.   NATURE IMMUNOLOGY