Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
(2016)
Journal Article
Hammond, S., Hazell, G., Shabanpoor, F., Saleh, A., Bowerman, M., Sleigh, J., …Wood, M. (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), 10962 - 10967. https://doi.org/10.1073/pnas.1605731113
The development of antisense oligonucleotide therapy is an important advance in the identification of corrective therapy for neuromuscular diseases, such as spinal muscular atrophy (SMA). Because of difficulties of delivering single-stranded oligonuc... Read More about Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy..