Restoration of human β-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides

by Thipparat Suwanmanee, Halin Sierakowska, Giuseppina Lacerra, Saovaros Svasti, Suzanne Kirby, Christopher E. Walsh, Suthat Fucharoen, Ryszard Kole
Published 2018

Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial

by Peter W. Collins, Guy Young, Karin Knobe, Faraizah Abdul Karim, Pantep Angchaisuksiri, Claus Banner, Türkiz Gürsel, Johnny Mahlangu, Tadashi Matsushita, Eveline P. Mauser-Bunschoten, Johannes Oldenburg, Christopher E. Walsh, Claude Negrier
Published 2018