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Molecular Therapy. Nucleic Acids|August 8, 2025
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challengesNechama Kalter, Carla Fuster-García, Alfredo Silva, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 24, 2024
Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responsesGiacomo Frati, Megane Brusson, Gilles Sartre, et al.
Cell Stem Cell|February 24, 2021
Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-SeqGiandomenico Turchiano, Geoffroy Andrieux, Julia Klermund, et al.
Nature Communications|August 14, 2020
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich SyndromeRajeev Rai, Marianna Romito, Elizabeth Rivers, et al.
Molecular Therapy. Methods & Clinical Development|October 10, 2022
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectorsMarta García, Jose Bonafont, Jesús Martínez-Palacios, et al.
The Journal of Allergy and Clinical Immunology|March 13, 2024
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemiaSameer Bahal, Marta Zinicola, Shefta E Moula, et al.
Nature Communications|June 19, 2026
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome modelsClaudiu A Cozmescu, Mina Nazari, Loukia Touramanidou, et al.
Nature Communications|November 3, 2025
Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCRNathan White, John Alexander Chalk, Yi-Ting Hu, et al.
Molecular Therapy. Nucleic Acids|November 30, 2023
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDiAlessia Cavazza, Ayal Hendel, Rasmus O Bak, et al.
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