Affiliation: University of Manchester
Cossu G, Previtali S, Napolitano S, Cicalese M, Tedesco F, Nicastro F, et al
. Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy. EMBO Mol Med. 2015;7:1513-28 pubmed publisher
..Intra-arterial transplantation of donor mesoangioblasts in human proved to be feasible and relatively safe. Future implementation of the protocol, together with a younger age of patients, will be needed to approach efficacy. ..
Benedetti S, Uno N, Hoshiya H, Ragazzi M, Ferrari G, Kazuki Y, et al
. Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy. EMBO Mol Med. 2018;10:254-275 pubmed publisher
..This work establishes a novel platform for complex gene transfer into clinically relevant human muscle progenitors for DMD gene therapy. ..
Galli F, Bragg L, Meggiolaro L, Rossi M, Caffarini M, Naz N, et al
. Gene and cell and therapy for muscular dystrophies: are we getting there?. Hum Gene Ther. 2018;: pubmed publisher
..It is thus a time of expectation, even though many hurdles remain and it is unclear now whether they will be solved by current strategies or further improvements will be necessary. ..
Aldeiri B, Roostalu U, Albertini A, Wong J, Morabito A, Cossu G. Transgelin-expressing myofibroblasts orchestrate ventral midline closure through TGF? signalling. Development. 2017;144:3336-3348 pubmed publisher