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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.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 19, 2009
Transcriptional enhancers induce insertional gene deregulation independently from the vector type and designGiulietta Maruggi, Simona Porcellini, Giulia Facchini, 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.
Frontiers in Neuroscience|April 11, 2022
Cerebellar Pathology in an Inducible Mouse Model of Friedreich AtaxiaElizabeth Mercado-Ayón, Nathan Warren, Sarah Halawani, et al.
Translational Research : the Journal of Laboratory and Clinical Medicine|July 30, 2017
Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primatesAlexandre Matet, Corinne Kostic, Alexis-Pierre Bemelmans, et al.
American Journal of Human Genetics|September 6, 2023
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathyGaia Andreoletti, Oriana Romano, Hsin-Jung Chou, et al.
Medical Sciences (Basel, Switzerland)|May 27, 2026
Breathing Under Pressure: Psychological Burden and Recovery Trajectories in Patients Receiving Non-Invasive Respiratory Support from Acute COVID-19 to Respiratory RehabilitationEleonora Volpato, Valentina Poletti, Maria Luisa de Candia, et al.
Human Gene Therapy|April 13, 2019
Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic AcidAlice Moussy, Nan Papili Gao, Guillaume Corre, et al.
Blood Advances|February 26, 2021
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cellsGiulia Pavani, Anna Fabiano, Marine Laurent, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 14, 2013
Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombinationAndrea Coluccio, Francesca Miselli, Angelo Lombardo, et al.
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