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Molecular Therapy. Methods & Clinical Development|August 25, 2018
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease PhenotypeLeslie Weber, Valentina Poletti, Elisa Magrin, et al.
Stem Cell Reports|February 11, 2014
Long-term stability and safety of transgenic cultured epidermal stem cells in gene therapy of junctional epidermolysis bullosaLaura De Rosa, Sonia Carulli, Fabienne Cocchiarella, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 21, 2018
CRISPR/Cas9-Mediated In Situ Correction of LAMB3 Gene in Keratinocytes Derived from a Junctional Epidermolysis Bullosa PatientDaniela Benati, Francesca Miselli, Fabienne Cocchiarella, et al.
American Journal of Physiology. Heart and Circulatory Physiology|April 30, 2026
The Medium Chain Fatty Acid Octanoate is a Beneficial Fuel for the Failing HeartNikoloz Gorgodze, Timothy R Matsuura, Noemi Nisini, et al.
EMBO Molecular Medicine|July 29, 2010
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patientsEmanuela Anna Roselli, Riccardo Mezzadra, Marta Claudia Frittoli, et al.
Scientific Reports|April 21, 2016
Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitmentOriana Romano, Clelia Peano, Guidantonio Malagoli Tagliazucchi, et al.
Molecular Therapy. Methods & Clinical Development|October 16, 2018
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-ThalassemiaMaria Rosa Lidonnici, Ylenia Paleari, Francesca Tiboni, et al.
Journal of Experimental & Clinical Cancer Research : CR|November 16, 2021
Alternative splicing of NF-YA promotes prostate cancer aggressiveness and represents a new molecular marker for clinical stratification of patientsSilvia Belluti, Valentina Semeghini, Giovanna Rigillo, et al.
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