Alessandro Aiuti

Vita-Salute San Raffaele University — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

79h-index34.6kcitations470works6.12yr avg
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Research Topics

Virus-based gene therapy research(162), CAR-T cell therapy research(105), Immunodeficiency and Autoimmune Disorders(81), RNA Interference and Gene Delivery(53), CRISPR and Genetic Engineering(52)

Publications462 total

Deleterious variants in the autophagy-related gene RB1CC1/FIP200 impair immunity to SARS-CoV-2
Nature Communications·2025· 1 citedOpen Access
Lentiviral hematopoietic stem and progenitor cell gene therapy for Wiskott-Aldrich Syndrome: Early access experience in Italian patients based on 648/1996 law
Blood·2025
Lentiviral hematopoietic stem and progenitor cell gene therapy with Etuvetidigene autotemcel for the treatment of Wiskott-Aldrich Syndrome
Blood·2025
Decoding the role of inflammatory signaling in shaping HSC heterogeneity and engraftment in gene therapy-treated β-thalassemia pediatric patients
Blood·2025
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2
Molecular Therapy — Methods & Clinical Development·2025· 1 citedOpen Access
Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome
Molecular Therapy·2025· 2 citedOpen Access
The seven enigmas of SARS-CoV-2: From the past to the future
Journal of Human Immunity·2025· 1 citedOpen Access
Autoantibodies neutralizing type I IFNs in 40% of patients with WNV encephalitis in seven new cohorts
2025Open Access
Ultrasonographic hip morphology in mucopolysaccharidosis type I Hurler after hematopoietic stem cell gene therapy
Journal of Children s Orthopaedics·2025Open Access
Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I
Molecular Therapy — Methods & Clinical Development·2025Open Access
Inborn errors of immunity: Manifestation, treatment, and outcome—an ESID registry 1994–2024 report on 30,628 patients
Journal of Human Immunity·2025· 10 citedOpen Access
Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia
Nature Communications·2025· 1 citedOpen Access
Considerations driving the choice in clinical trial design of cell and gene therapy products: weighing convenience versus necessity
Cytotherapy·2025· 2 citedOpen Access
Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET)
EClinicalMedicine·2025Open Access
In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking
Nature·2025· 9 citedOpen Access
The View from a Milestone in Gene Therapy
New England Journal of Medicine·2025· 2 cited
Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy
Brain·2025· 1 cited
Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program
International Journal of Neonatal Screening·2025Open Access
Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy
New England Journal of Medicine·2025· 14 cited
Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID
Molecular Therapy·2025
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Frequent Co-authors

Fabio Ciceri(15), Maria Grazia Roncarolo(13), Luigi Naldini(12), Luigi D. Notarangelo(10), Claudio Bordignon(10), Giorgio Casari(9), Maria Pia Cicalese(9), Federica Barzaghi(9), Paul Bastard(7), Filomeen Haerynck(7), Andrés A. Arias(7), Luca Biasco(7), Samantha Scaramuzza(7), José Luis Franco(7), Alexandre Bélot(6), Lluís Quintana‐Murci(6), Stuart G. Tangye(6), Petter Brodin(6), Carlos Rodríguez‐Gallego(6), Eugenio Montini(6)