Hammond, SM;
Hazell, G;
Shabanpoor, F;
Saleh, AF;
Bowerman, M;
Sleigh, JN;
Meijboom, KE;
... Wood, MJ; + view all
(2016)
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Proceedings of the National Academy of Sciences of the United States of America
, 113
(39)
pp. 10962-10967.
10.1073/pnas.1605731113.