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The study, co-authored by Jin Jiongzhu (transliteration), was published in the online edition of the Journal of Clinical Investigation Insights on August 9, 2022, with the title "A pathogenic proteolysis-resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function.
Huntington's disease is a dominantly inherited neurodegenerative disorder caused by mutations in a protein called "huntingtin," which increases a protein called "polyglutamine.
Cleavage near stretched polyglutamine in mutant huntingtin is thought to be responsible for Huntington's disease
This work was supported by a Global Research Laboratory grant from the National Research Foundation of Korea (NRF) and a EUREKA Eurostars 2 grant from EU Horizon 2020
Journal Reference:
Hyeongju Kim, Sophie Lenoir, Angela Helfricht, Taeyang Jung, Zhana K.