Open AccessSemirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders
Author(s) -
Jihad El Andari,
Edith Renaud-Gabardos,
Warut Tulalamba,
Jonas Weinmann,
Louise Mangin,
Quang Hong Pham,
Susanne Hille,
Antonette Bennett,
Esther Attebi,
Emanuele Bourges,
Christian Leborgne,
Nicolas Guerchet,
Julia Fakhiri,
Chiara Krämer,
Ellen Wiedtke,
Robert McKenna,
Laurence Guianvarc’h,
Magali Toueille,
Giuseppe Ronzitti,
Matthias Hebben,
Federico Mingozzi,
Thierry VandenDriessche,
Mavis AgbandjeMcKenna,
Oliver Müller,
Marinee Chuah,
Ana BujBello,
Dirk Grimm
Publication year - 2022
Publication title -
science advances
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.928
H-Index - 146
ISSN - 2375-2548
DOI - 10.1126/sciadv.abn4704
Subject(s) - capsid , genetic enhancement , duchenne muscular dystrophy , computational biology , gene delivery , muscle disorder , skeletal muscle , biology , mdx mouse , gene , adeno associated virus , muscular dystrophy , vector (molecular biology) , bioinformatics , genome editing , viral vector , dystrophin , microbiology and biotechnology , virology , medicine , genome , recombinant dna , genetics , anatomy