BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
Strings-Ufombah, Vanessa, Malerba, Alberto, Kao, Shih-Chu, Harbaran, Sonal, Roth, Fanny, Cappellari, Ornella, Lu-Nguyen, Ngoc, Takahashi, Keiko, Mukadam, Sophie, Kilfoil, Georgina, Kloth, Claudia, Roelvink, Petrus, Dickson, George, Trollet, Capucine, Suhy, David
Published in Molecular therapy. Nucleic acids (04.06.2021)
Published in Molecular therapy. Nucleic acids (04.06.2021)
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Journal Article
비-기능성 T-세포 수용체 (TCR)를 갖는 T-세포를 생산하기 위한 시약, 이를 포함하는 조성물 및 이의 용도
GRAHAM MICHAEL, SUHY DAVID, STRINGS UFOMBAH VANESSA, ROELVINK PETER, GARCIA PATTY BERTHA
Year of Publication 10.06.2019
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Year of Publication 10.06.2019
Patent
Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1
Malerba, Alberto, Klein, Pierre, Lu-Nguyen, Ngoc, Cappellari, Ornella, Strings-Ufombah, Vanessa, Harbaran, Sonal, Roelvink, Peter, Suhy, David, Trollet, Capucine, Dickson, George
Published in Human molecular genetics (01.10.2019)
Published in Human molecular genetics (01.10.2019)
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Journal Article