Cristina Mesa-Núñez

The San Raffaele Telethon Institute for Gene Therapy — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

6h-index195citations18works2.72yr avg
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Research Topics

Virus-based gene therapy research(7), Immunodeficiency and Autoimmune Disorders(5), Cell Adhesion Molecules Research(4), Blood disorders and treatments(4), CAR-T cell therapy research(3)

Publications18 total

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
Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities
Journal of Allergy and Clinical Immunology·2024· 6 citedOpen Access
Early Bone Marrow Alterations in Patients with Adenosine Deaminase 2 Deficiency Across Disease Phenotypes and Severities
SSRN Electronic Journal·2024· 1 citedOpen Access
Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study
Transplantation and Cellular Therapy·2023· 1 cited
Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated <i>Ex-vivo</i> Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Blood·2022· 2 cited
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I
Molecular Therapy — Methods & Clinical Development·2022· 6 citedOpen Access
A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
Blood·2021· 6 cited
A novel genetic variant in <scp><i>PTGS1</i></scp> affects N‐glycosylation of cyclooxygenase‐1 causing a dominant‐negative effect on platelet function and bleeding diathesis
American Journal of Hematology·2020· 4 citedOpen Access
A Phase 1/2 Study of Lentiviral-Mediated <i>Ex-Vivo</i> Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1
Blood·2020· 12 cited
The downregulated membrane expression of CD18 in CD34+ cells defines a primitive population of human hematopoietic stem cells
Stem Cell Research & Therapy·2020· 5 citedOpen Access
A New Molecular Variant in the PTGS1 Gene That Abrogates Generation of Thromboxane A2 Synthesis and Associates with Platelet Dysfunction and Bleeding
Blood·2019
Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions
Blood·2019· 4 cited
Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I
Blood·2018
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors
Molecular Therapy·2018· 70 citedOpen Access
In vivo imaging of lung inflammation with neutrophil-specific 68Ga nano-radiotracer
Scientific Reports·2017· 52 citedOpen Access
A Short and Efficient Transduction Protocol for Mouse Hematopoietic Stem Cells with Lentiviral Vectors
Human Gene Therapy Methods·2017· 3 cited
Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I
Human Gene Therapy·2016· 22 citedOpen Access
Biotech Annual Congress 2013 - VII Congreso de la Federacion Española de Biotecnologia
2013

Frequent Co-authors

Elena Almarza(14), Juan A. Bueren(13), Julián Sevilla(5), Adrian J. Thrasher(4), Donald B. Kohn(4), Dayna Terrazas(4), Eileen Nicoletti(4), Augustine Fernandes(4), Caroline Y. Kuo(4), Satiro De Oliveira(4), Theodore B. Moore(4), Jonathan D. Schwartz(4), Carlos Damián(4), María Fernández-García(4), Brian C. Beard(3), Begoña Díez(3), Jonathan Schwartz(3), Rosa María Pérez Yáñez(3), Dimitri Bulté(3), Federica Barzaghi(3)