Not logged inSevere inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Sobrino, Steicy; Joseph, Laure; Magrin, Elisa; Chalumeau, Anne; Hebert, Nicolas; Corsia, Alice; Denis, Adeline; Roudaut, Cecile; Aussel, Clotilde; Leblanc, Olivia; Brusson, Megane; Felix, Tristan; Diana, Jean-Sebastien; Petrichenko, Angelina; El Etri, Jana; Godard, Auria; Tibi, Eden; Manceau, Sandra; Treluyer, Jean Marc; Mavilio, Fulvio; Bushman, Frederic D.; Marcais, Ambroise; Castelle, Martin; Neven, Benedicte; Hermine, Olivier; Renolleau, Sylvain; Magnani, Alessandra; Asnafi, Vahid; El Nemer, Wassim; Bartolucci, Pablo; Six, Emmanuelle; Semeraro, Michaela; Miccio, Annarita .png)
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SaveCorrection of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe diseaseWantucha, Slawomir; Tuskeb, Steven; Benedettic, Sara; Recchia, Chiara; Heala, Paul; Sagooa, Pervinder; Johnsonb, Tyler; Mehtab, Nickita; Brudvigb, Jon; Mavilioa, Fulvio; Weimerb, Jill; Gaspard, Hubert Bobby Share Save High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathyAndreoletti, Gaia; Romano, Oriana; Chou, Hsin-Jung; Sefid-Dashti, Mahjoubeh J.; Grilli, Andrea; Chen, Clarice; Lakshman, Neema; Purushothaman, Pravin; Varfaj, Fatbardha; Mavilio, Fulvio; Bicciato, Silvio; Urbinati, Fabrizia Share Save Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategiesBrusson, Megane; Chalumeau, Anne; Martinucci, Pierre; Romano, Oriana; Felix, Tristan; Poletti, Valentina; Scaramuzza, Samantha; Ramadier, Sophie; Masson, Cecile; Ferrari, Giuliana; Mavilio, Fulvio; Cavazzana, Marina; Amendola, Mario; Miccio, Annarita Share Save Share Save Cerebellar Pathology in an Inducible Mouse Model of Friedreich AtaxiaMercado-Ayon, Elizabeth; Warren, Nathan; Halawani, Sarah; Rodden, Layne N.; Ngaba, Lucie; Dong, Yi Na; Chang, Joshua C.; Fonck, Carlos; Mavilio, Fulvio; Lynch, David R.; Lin, Hong Share Save Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicityEggers, Michelle; Vannoy, Charles H.; Huang, Jianyong; Purushothaman, Pravinkumar; Brassard, Jacqueline; Fonck, Carlos; Meng, Hui; Prom, Mariah J.; Lawlor, Michael W.; Cunningham, Justine; Sadhu, Chanchal; Mavilio, Fulvio Share Save Share Save Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cellsPavani, Giulia; Fabiano, Anna; Laurent, Marine; Amor, Fatima; Cantelli, Erika; Chalumeau, Anne; Maule, Giulia; Tachtsidi, Alexandra; Concordet, Jean-Paul; Cereseto, Anna; Mavilio, Fulvio; Ferrari, Giuliana; Miccio, Annarita; Amendola, Mario Share Save Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotypeWeber, Leslie; Frati, Giacomo; Felix, Tristan; Hardouin, Giulia; Casini, Antonio; Wollenschlaeger, Clara; Meneghini, Vasco; Masson, Cecile; De Cian, Anne; Chalumeau, Anne; Mavilio, Fulvio; Amendola, Mario; Andre-Schmutz, Isabelle; Cereseto, Anna; El Nemer, Wassim; Concordet, Jean-Paul; Giovannangeli, Carine; Cavazzana, Marina; Miccio, Annarita Share Save Pre-clinical safety and efficacy findings of AT845, a novel gene replacement therapy for Pompe disease targeting skeletal muscle and heartMavilio, Fulvio; Cunningham, Justine; Eggers, Michelle; Heffner, Garrett; Stinchcombe, Tim; Brassard, Jacqueline; Chaivorapol, Christina; Gray, John; Prasad, Suyash; James, Emma; Rico, Salvador Share Save Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic RearrangementsLattanzi, Annalisa; Meneghini, Vasco; Pavani, Giulia; Amor, Fatima; Ramadier, Sophie; Felix, Tristan; Antoniani, Chiara; Masson, Cecile; Alibeu, Olivier; Lee, Ciaran; Porteus, Matthew H.; Bao, Gang; Amendola, Mario; Mavilio, Fulvio; Miccio, Annarita Share Save Share Save Share Save Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon VectorsHolstein, Marta; Mesa-Nunez, Cristina; Miskey, Csaba; Almarza, Elena; Poletti, Valentina; Schmeer, Marco; Grueso, Esther; Flores, Juan Carlos Ordonez; Kobelt, Dennis; Walther, Wolfgang; Aneja, Manish K.; Geiger, Johannes; Bonig, Halvard B.; Izsvak, Zsuzsanna; Schleef, Martin; Rudolph, Carsten; Mavilio, Fulvio; Bueren, Juan A.; Guenechea, Guillermo; Ivics, Zoltan Share Save Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiencySoheili, Tayebeh; Durand, Amandine; Sepulveda, Fernando E.; Riviere, Julie; Lagresle-Peyrou, Chantal; Sadek, Hanem; de Saint Basile, Genevieve; Martin, Samia; Mavilio, Fulvio; Cavazzana, Marina; Andre-Schmutz, Isabelle Share Save Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primatesMatet, Alexandre; Kostic, Corinne; Bemelmans, Alexis-Pierre; Moulin, Alexandre; Rosolen, Serge G.; Martin, Samia; Mavilio, Fulvio; Amirjanians, Vazrik; Stieger, Knut; Lorenz, Birgit; Behar-Cohen, Francine; Arsenijevic, Yvan Share Save Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophyLe Guiner, Caroline; Servais, Laurent; Montus, Marie; Larcher, Thibaut; Fraysse, Bodvael; Moullec, Sophie; Allais, Marine; Francois, Virginie; Dutilleul, Maeva; Malerba, Alberto; Koo, Taeyoung; Thibaut, Jean-Laurent; Matot, Beatrice; Devaux, Marie; Le Duff, Johanne; Deschamps, Jack-Yves; Barthelemy, Ines; Blot, Stephane; Testault, Isabelle; Wahbi, Karim; Ederhy, Stephane; Martin, Samia; Veron, Philippe; Georger, Christophe; Athanasopoulos, Takis; Masurier, Carole; Mingozzi, Federico; Carlier, Pierre; Gjata, Bernard; Hogrel, Jean-Yves; Adjali, Oumeya; Mavilio, Fulvio; Voit, Thomas; Moullier, Philippe; Dickson, George Share Save Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 SystemLattanzi, Annalisa; Duguez, Stephanie; Moiani, Arianna; Izmiryan, Araksya; Barbon, Elena; Martin, Samia; Mamchaoui, Kamel; Mouly, Vincent; Bernardi, Francesco; Mavilio, Fulvio; Bovolenta, Matteo Share Save Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in DogsMack, David L.; Poulard, Karine; Goddard, Melissa A.; Latoumerie, Virginie; Snyder, Jessica M.; Grange, Robert W.; Elverman, Matthew R.; Denard, Jerome; Veron, Philippe; Buscara, Laurine; Le Bec, Christine; Hogrel, Jean-Yves; Brezovec, Annie G.; Meng, Hui; Yang, Lin; Liu, Fujun; O'Callaghan, Michael; Gopal, Nikhil; Kelly, Valerie E.; Smith, Barbara K.; Strande, Jennifer L.; Mavilio, Fulvio; Beggs, Alan H.; Mingozzi, Federico; Lawlor, Michael W.; Buj-Bello, Ana; Childers, Martin K. 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