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Image: Anti-sense oligotide (AON)-induced huntingtin protein is resistant to the cleavage of Caspase-6, so it does not cause Huntington's disease
ProQR Therapeutics in the Netherlands, Université Grenoble Alps in France, KTH Royal Institute of Technology in Sweden and other international joint research, Song Ji-soon Research Group of the Department of Life Sciences of the Korean Academy of Sciences (KAIST) and KAIST Biological Century Institute jointly formulated a noble strategy
The study, titled "Antisense oligonucleotide-induced huntingtin subtypes with pathogenic proteolytic resistance to maintain huntingtin function," was co-authored by Hyeongju Kim and published in the online edition of the Journal of Clinical Investigation Insight on August 9, 2022
Huntington's disease is a dominant hereditary neurodegenerative disease caused by a mutation in a protein called "huntingtin" that adds an extended extension of a glutamine amino acid called "polyglutamine" to the protein, which is a unique feature
Cleavage near stretched polyglutamine in the mutant huntingtin protein is thought to be the cause of
This study was welcomed because it would certainly drive innovative strategies to address Huntington's disease without altering the basic function
This work was supported by a Global Research Laboratory grant from the Korea National Research Foundation (NRF) and a EUREKA Eurostars 2 grant from EU Horizon 2020
