Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

Exon skipping using phosphorodiamidate morpholino oligomers (PMOs) is a promising treatment strategy for Duchenne muscular dystrophy (DMD). The most significant limitation of these clinically used compounds is their lack of delivery systems that target muscles; thus, cell-penetrating peptides are be...

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Detalles Bibliográficos
Autores principales: Miyatake, Shouta, Mizobe, Yoshitaka, Tsoumpra, Maria K., Lim, Kenji Rowel Q., Hara, Yuko, Shabanpoor, Fazel, Yokota, Toshifumi, Takeda, Shin’ichi, Aoki, Yoshitsugu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6374502/
https://www.ncbi.nlm.nih.gov/pubmed/30763772
http://dx.doi.org/10.1016/j.omtn.2019.01.008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6374502/
https://www.ncbi.nlm.nih.gov/pubmed/30763772
http://dx.doi.org/10.1016/j.omtn.2019.01.008

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