A joint research team, consisting of researchers from Korea, Netherlands, France, and Sweden, said Friday that it came up with a new method to treat Huntington's disease (HD).

A Korean and European research team, participated by Professor Song Ji-joon at the Korea Advanced Institute of Science and Technology, has discovered a new clue in treating Huntington's disease.
A Korean and European research team, participated by Professor Song Ji-joon at the Korea Advanced Institute of Science and Technology, has discovered a new clue in treating Huntington's disease.

HD is a rare genetic neurodegenerative disease accompanied by loss of coordination between muscles, cognitive decline, and psychological problems. It occurs in one per 10,000 to 30,000 people and leads to death after about a decade of degenerative processes.

As an inherited degenerative brain illness, HD is caused by a mutation in the huntingtin protein, which is a large protein of more than 3,000 connected amino acids.

While the huntingtin protein causes the disease, it is also essential for biological functions, which, in turn, makes it important to select only disease-causing proteins as therapeutic targets.

The research team, consisting of Professor Song Ji-joon at the Korea Advanced Institute of Science and Technology (KAIST), ProQR Therapeutics, a Dutch-based biopharmaceutical company, researchers from France's Université Grenoble Alpes, and Sweden's KTH Royal Institute of Technology, gathered to find a new HD therapeutic strategy.

The results showed that researchers could use a new method of converting the mutant Huntingtin protein into a disease-free form while maintaining its original function.

The research team found that the huntingtin delta 12 form, produced using antisense oligonucleotide, a type of RNA, was resistant to caspase-6 cleavage. Still, the huntingtin protein retained overall biochemical and structural properties.

The team expects that the result of its study will be applicable in developing a treatment for HD.

"This study was conducted through joint research in four countries, including Korea, and we expect that the method of inducing the disease-causing huntingtin protein to a normal state will open a new path for developing HD therapy," Song said.

Journal of Clinical Investigation Insights published the result of the study in its online edition.

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