Blar i forfatter Det helsevitenskapelige fakultet "Aartsma-Rus, Annemieke"

Viser treff 1-1 av 1

    • Delivery is key: lessons learnt from developing splice-switching antisense therapies 

      Godfrey, Caroline; Desviat, Lourdes R.; Smedsrød, Bård; Piétri-Rouxel, France; Denti, Michela A.; Disterer, Petra; Lorain, Stéphanie; Nogales-Gadea, Gisela; Sardone, Valentina; Anwar, Rayan; El Andaloussi, Samir; Letho, Taavi; Khoo, Bernard; Brolin, Camilla; Mc Van Roon-Mom, Willeke; Goyenvalle, Aurélie; Aartsma-Rus, Annemieke; Arechavala-Gomeza, Virginia (Journal article; Tidsskriftartikkel; Peer reviewed, 2017-03-13)
      The use of splice-switching antisense therapy is highly promising, with a wealth of pre-clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The main obstacle impeding the clinical translation of this approach is the relatively poor delivery of anti- sense oligonucleotides to target tissues ...