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Human Gene Therapy|September 11, 2018
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison StudyFabrizia Urbinati, Beatriz Campo Fernandez, Katelyn E Masiuk, et al.
Virology|March 26, 2013
Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replicationFrancesca Di Nunzio, Thomas Fricke, Annarita Miccio, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 25, 2008
Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectorsFrancesca Di Nunzio, Giulietta Maruggi, Stefano Ferrari, et al.
Veterinary Sciences|August 28, 2024
Generation of Insulin-Producing Cells from Canine Bone Marrow-Derived Mesenchymal Stem Cells: A Preliminary StudyAntonella Colella, Giuseppina Biondi, Nicola Marrano, et al.
Molecular Therapy. Methods & Clinical Development|September 22, 2016
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophyNicole Armbruster, Annalisa Lattanzi, Matthieu Jeavons, et al.
Blood|September 25, 2010
High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitorsClaudia Cattoglio, Danilo Pellin, Ermanno Rizzi, et al.
Molecular Therapy. Nucleic Acids|November 23, 2016
In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse RetinaMaria Carmela Latella, Maria Teresa Di Salvo, Fabienne Cocchiarella, et al.
Microorganisms|August 29, 2024
A Pilot Study for the Characterization of Bacillus spp. and Analysis of Possible B. thuringiensis/Strongyloides stercoralis CorrelationElena Pomari, Pierantonio Orza, Milena Bernardi, et al.
EMBO Molecular Medicine|December 1, 2021
Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicityMichelle Eggers, Charles H Vannoy, Jianyong Huang, et al.
Blood Advances|January 4, 2018
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiencyTayebeh Soheili, Amandine Durand, Fernando E Sepulveda, et al.
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