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Nature Biotechnology|June 1, 2026
Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reportersDaniele Canarutto, Martina Fiumara, Vigneshwaran Venkatesan, et al.
Leukemia|August 28, 2023
miR-126 identifies a quiescent and chemo-resistant human B-ALL cell subset that correlates with minimal residual diseaseCarolina Caserta, Silvia Nucera, Matteo Barcella, et al.
The Journal of Pediatrics|September 21, 2021
Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring SystemSilvia Pontesilli, Cristina Baldoli, Pasquale Anthony Della Rosa, et al.
Nature Communications|March 8, 2023
Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemiaMatteo Maria Naldini, Gabriele Casirati, Matteo Barcella, et al.
Blood|December 14, 2017
Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infectionsMiriam Hetzel, Adele Mucci, Patrick Blank, et al.
Blood|June 23, 2017
NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host diseaseSara Mastaglio, Pietro Genovese, Zulma Magnani, et al.
Molecular Therapy. Methods & Clinical Development|September 6, 2021
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year seriesDaniele Canarutto, Francesca Tucci, Salvatore Gattillo, et al.
Cell Reports. Medicine|November 13, 2024
A p38 MAPK-ROS axis fuels proliferation stress and DNA damage during CRISPR-Cas9 gene editing in hematopoietic stem and progenitor cellsLucrezia Della Volpe, Federico Midena, Roberta Vacca, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 27, 2026
Ex Vivo Expansion of Hematopoietic Stem and Progenitor Cells from Human Mobilized Peripheral Blood for Gene Therapy ApplicationsErika Zonari, Matteo Maria Naldini, Matteo Barcella, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 3, 2020
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene TherapyRaisa Jofra Hernández, Andrea Calabria, Francesca Sanvito, et al.
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