Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat

Friedreich's Ataxia (FA) is an inherited neurologic disorder caused by an expanded GAA repeat within intron 1 of the frataxin (FXN) gene that reduces expression of FXN protein. Agents that increase expression of FXN have the potential to alleviate the disease. We previously reported that duplex...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Nucleic Acid Ther
Main Authors: Li, Liande, Shen, Xiulong, Liu, Zhongtian, Norrbom, Michaela, Prakash, Thazha P., O'Reilly, Daniel, Sharma, Vivek K., Damha, Masad J., Watts, Jonathan K., Rigo, Frank, Corey, David R.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5790436/
https://ncbi.nlm.nih.gov/pubmed/29341839
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2017.0703
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados