2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring hemoglobin A production and chain rebalance

β-thalassemia is a disorder caused by altered hemoglobin protein synthesis which affects individuals worldwide. Severe forms of the disease, left untreated, can result in death before the age of 3 years.(1) The standard of care consists of chronic and costly palliative treatment by blood transfusion...

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Detalles Bibliográficos
Autores principales: Dong, Alisa, Ghiaccio, Valentina, Motta, Irene, Guo, Shuling, Peralta, Raechel, Freier, Susan M., Watt, Andy, Damle, Sagar, Ikawa, Yasuhiro, Jarocha, Danuta, Chappell, Maxwell, Stephanou, Coralea, Delbini, Paola, Chen, Connie, Christou, Soteroula, Kleanthous, Marina, Smith-Whitley, Kim, Manwani, Deepa, Casu, Carla, Abdulmalik, Osheiza, Cappellini, Maria Domenica, Rivella, Stefano, Breda, Laura
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Fondazione Ferrata Storti 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8094087/
https://www.ncbi.nlm.nih.gov/pubmed/32439726
http://dx.doi.org/10.3324/haematol.2019.226852

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8094087/
https://www.ncbi.nlm.nih.gov/pubmed/32439726
http://dx.doi.org/10.3324/haematol.2019.226852

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