Claudia Rossi

The San Raffaele Telethon Institute for Gene Therapy — Italy

Biochemistry, Genetics and Molecular Biology · Life Sciences

16h-index2.0kcitations34works0.32yr avg
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Research Topics

Virus-based gene therapy research(9), CRISPR and Genetic Engineering(9), Hemoglobinopathies and Related Disorders(6), Neurotransmitter Receptor Influence on Behavior(4), Neuroscience and Neuropharmacology Research(3)

Publications34 total

Decoding the role of inflammatory signaling in shaping HSC heterogeneity and engraftment in gene therapy-treated β-thalassemia pediatric patients
Blood·2025
Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia
Nature Communications·2025· 1 citedOpen Access
3117 – HSCS IN Β-THALASSEMIA HAVE A REDUCED DORMANCY AND AN ERYTHROID BIASED LINEAGE COMMITMENT IN RESPONSE TO ALTERED TGFΒ SIGNALING IN THE BM MICROENVIRONMENT
Experimental Hematology·2024
S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY
HemaSphere·2022Open Access
Hematopoietic Stem Cells Are Endowed with Erythroid Signature in Beta-Thalassemia
Blood·2020
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
Nature Medicine·2019· 222 cited
Achieving orphan designation for placental insufficiency: annual incidence estimations in Europe
BJOG An International Journal of Obstetrics & Gynaecology·2018· 24 cited
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
Molecular Therapy — Methods & Clinical Development·2018· 23 citedOpen Access
Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector
Blood·2017· 13 cited
Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment
Scientific Reports·2016· 21 citedOpen Access
MATERNAL UTERINE ARTERY GENE THERAPY WITH AD.VEGF-A(165) INCREASES WEIGHT AT TERM IN A GUINEA PIG MODEL OF FETAL GROWTH RESTRICTION
UCL Discovery (University College London)·2015
Preclinical safety studies for gene therapy medicinal products in GLP test facility
Toxicology Letters·2014
The GATA1-HS2 Enhancer Allows Persistent and Position-Independent Expression of a β-globin Transgene
PLoS ONE·2011· 24 citedOpen Access
MicroRNA and proliferation control in chronic lymphocytic leukemia: functional relationship between miR-221/222 cluster and p27
Blood·2010· 110 citedOpen Access
<i>In vivo</i> selection of genetically modified erythroblastic progenitors leads to long-term correction of β-thalassemia
Proceedings of the National Academy of Sciences·2008· 149 citedOpen Access
Lentiviral-transduced HSC lead to long-term correction of beta-thalassemia by in vivo selection or erythroid progenitors.
Molecular Therapy·2007
Small for gestational age and Fetal Growth Restricted (FGR) very low birth weight neonates have different postnatal weight growth curves
2007
667. Gene Therapy for Beta Thalassemia: Preclinical Studies on Human Cells
Molecular Therapy·2006Open Access
70. Long Term Correction of beta-Thalassemia by Transplantation of Transduced Hematopoietic Stem Cells
Molecular Therapy·2006Open Access
Polyomavirus BK infection in pediatric kidney-allograft recipients: a single-center analysis of incidence, risk factors, and novel therapeutic approaches
Transplantation·2003· 201 cited
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Frequent Co-authors

Giuliana Ferrari(19), Sarah Marktel(8), Maria Rosa Lidonnici(8), Alessandro Aiuti(8), Francesca Tiboni(8), Samantha Scaramuzza(7), Fabio Ciceri(7), Fulvio Mavilio(6), Luigi Naldini(6), Annarita Miccio(6), Andrea Calabria(5), Eugenio Montini(5), Francesca Sanvito(5), Luigi Cervo(5), R. Samanin(5), Maria Ester Bernardo(4), Rossano Cesari(4), Michael Antoniou(4), Giulia Chianella(4), Matteo Barcella(4)