Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

Por Shabanpoor, Fazel, Hammond, Suzan M, Abendroth, Frank, Hazell, Gareth, Wood, Matthew J.A., Gait, Michael J.
Publicado no Nucleic Acid Ther (2017)

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Por Hammond, Suzan M., Hazell, Gareth, Shabanpoor, Fazel, Saleh, Amer F., Bowerman, Melissa, Sleigh, James N., Meijboom, Katharina E., Zhou, Haiyan, Muntoni, Francesco, Talbot, Kevin, Gait, Michael J., Wood, Matthew J. A.
Publicado no Proc Natl Acad Sci U S A (2016)