未登录Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease严重炎症和谱系偏移与 sickle cell disease 患者中工程化造血干细胞的植入不良相关。
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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收藏Correction 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 分享 收藏 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 分享 收藏 Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies结合基因添加和基因沉默策略的镰状细胞病基因治疗的新型慢病毒载体Brusson, 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 分享 收藏 分享 收藏 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 分享 收藏 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 分享 收藏 分享 收藏 Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells通过CRISPR/Cas9编辑人类造血干细胞中的 α-珠蛋白基因座来校正 β-地中海贫血Pavani, 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 分享 收藏 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 分享 收藏 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 分享 收藏 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 分享 收藏 分享 收藏 分享 收藏 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 分享 收藏 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 分享 收藏 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 分享 收藏 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 分享 收藏 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 分享 收藏 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. 分享 收藏 
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