BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

Vanessa Strings-Ufombah; Alberto Malerba; Shih-Chu Kao; Sonal Harbaran; Fanny Roth; Ornella Cappellari; Ngoc Lu-Nguyen; Keiko Takahashi; Sophie Mukadam; Georgina Kilfoil; Claudia Kloth; Petrus Roelvink; George Dickson; Capucine Trollet; David Suhy

doi:10.1016/j.omtn.2021.02.017

BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

Vanessa Strings-Ufombah, Alberto Malerba, Shih-Chu Kao, Sonal Harbaran, Fanny Roth, Ornella Cappellari, Ngoc Lu-Nguyen, Keiko Takahashi, Sophie Mukadam, Georgina Kilfoil, Claudia Kloth, Petrus Roelvink, George Dickson, Capucine Trollet, David Suhy

Research output: Contribution to journal › Article › peer-review

Original language	English
Pages (from-to)	67-78
Number of pages	12
Journal	Molecular therapy. Nucleic acids
Volume	24
Early online date	18 Feb 2021
DOIs	https://doi.org/10.1016/j.omtn.2021.02.017
Publication status	Published - 4 Jun 2021

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10.1016/j.omtn.2021.02.017Licence: CC BY-NC-ND

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Strings-Ufombah, V., Malerba, A., Kao, S.-C., Harbaran, S., Roth, F., Cappellari, O., Lu-Nguyen, N., Takahashi, K., Mukadam, S., Kilfoil, G., Kloth, C., Roelvink, P., Dickson, G., Trollet, C., & Suhy, D. (2021). BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy. Molecular therapy. Nucleic acids, 24, 67-78. https://doi.org/10.1016/j.omtn.2021.02.017

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title = "BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy",

author = "Vanessa Strings-Ufombah and Alberto Malerba and Shih-Chu Kao and Sonal Harbaran and Fanny Roth and Ornella Cappellari and Ngoc Lu-Nguyen and Keiko Takahashi and Sophie Mukadam and Georgina Kilfoil and Claudia Kloth and Petrus Roelvink and George Dickson and Capucine Trollet and David Suhy",

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Strings-Ufombah, V, Malerba, A, Kao, S-C, Harbaran, S, Roth, F, Cappellari, O, Lu-Nguyen, N, Takahashi, K, Mukadam, S, Kilfoil, G, Kloth, C, Roelvink, P, Dickson, G, Trollet, C & Suhy, D 2021, 'BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy', Molecular therapy. Nucleic acids, vol. 24, pp. 67-78. https://doi.org/10.1016/j.omtn.2021.02.017

TY - JOUR

T1 - BB-301

T2 - a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

AU - Strings-Ufombah, Vanessa

AU - Malerba, Alberto

AU - Kao, Shih-Chu

AU - Harbaran, Sonal

AU - Roth, Fanny

AU - Cappellari, Ornella

AU - Lu-Nguyen, Ngoc

AU - Takahashi, Keiko

AU - Mukadam, Sophie

AU - Kilfoil, Georgina

AU - Kloth, Claudia

AU - Roelvink, Petrus

AU - Dickson, George

AU - Trollet, Capucine

AU - Suhy, David

PY - 2021/6/4

Y1 - 2021/6/4

U2 - 10.1016/j.omtn.2021.02.017

DO - 10.1016/j.omtn.2021.02.017

M3 - Article

C2 - 33738139

SN - 2162-2531

VL - 24

SP - 67

EP - 78

JO - Molecular therapy. Nucleic acids

JF - Molecular therapy. Nucleic acids

ER -