Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.

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Detalles Bibliográficos
Autores principales: Yuanjing Liu, Jean-Cosme Dodart, Helene Tran, Shaunna Berkovitch, Maurine Braun, Michael Byrne, Ann F. Durbin, Xiao Shelley Hu, Naoki Iwamoto, Hyun Gyung Jang, Pachamuthu Kandasamy, Fangjun Liu, Kenneth Longo, Jörg Ruschel, Juili Shelke, Hailin Yang, Yuan Yin, Amy Donner, Zhong Zhong, Chandra Vargeese, Robert H. Brown
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Science
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Acceso en línea:https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de
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Sumario:C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.

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