Towards Personalized Allele-Specific Antisense Oligonucleotide Therapies for Toxic Gain-of-Function Neurodegenerative Diseases

por Helm, Jacob, Schöls, Ludger, Hauser, Stefan
Publicado 2022

Allele-specific targeting of mutant ataxin-3 by antisense oligonucleotides in SCA3-iPSC-derived neurons

por Hauser, Stefan, Helm, Jacob, Kraft, Melanie, Korneck, Milena, Hübener-Schmid, Jeannette, Schöls, Ludger
Publicado 2021