New solution to treat hereditary disease 'Huntington's disease'

Huntington’s disease resistance huntingtin protein induced by antisense oligonucleotide (AON) is not cleaved by Caspase-6, so it has resistance to Huntington’s disease and at the same time maintains the normal function of huntingtin. [사진=KAIST]

In a situation where there is no cure for Huntington’s disease, an inherited degenerative brain disease, a domestic research team has opened the way to develop a treatment.

KAIST (President Lee Kwang-hyung) announced on the 4th that a research team led by Professor Ji-Jun Song of the Department of Life Sciences presented a new concept method to treat Huntington’s disease.

Huntington’s disease is a rare genetic disease, a neurodegenerative disease that is accompanied by loss of coordination between muscles, cognitive decline, and psychological problems. It is an inherited degenerative brain disease and is caused by a mutation in the Huntington protein that expands several glutamine amino acids in succession.

The disease has an incidence of about 1 in 10,000 to 30,000 people. It is a disease that leads to death after 10 years of degenerative process. Huntingtin protein, a giant protein made by linking more than 3,000 amino acids, causes disease, but is an essential protein for biological functions. Therefore, it is very important to select only disease-causing proteins as therapeutic targets.

Accordingly, the research team proposed a new methodology for treating Huntington’s disease by converting the mutant huntingtin protein that causes Huntington’s disease into a disease-free form while maintaining its original function. The form of huntingtin delta 12, induced by using an antisense oligonucleotide, a type of RNA, is not cut due to the amino acid terminus of the protein, which is the main cause of Huntington’s disease. stated that the function was maintained.

The research team predicted that the results of this study could be applied to developing a treatment for Huntington’s disease. Professor Song Ji-jun said, “This study is expected to open a new path for the development of a treatment for Huntington’s disease by inducing the disease-causing huntingtin protein to a normal state.”

Meanwhile, this research was carried out with support from the Global Research Laboratory project of the National Research Foundation of Korea. The research results were published in the international academic journal ‘Journal of Clinical Investigation Insights’.