Delivery times 2024-01-24 15:49
“Reduction of lower limb stiffness and improvement of gait disturbances in animal experiments”
Schematic diagram verifying the effectiveness of the treatment
[한국생명공학연구원 제공. 재판매 및 DB 금지]
(Daejeon = Yonhap News) Reporter Chan-wook Jeong = The Korea Bioscience and Biotechnology Research Institute announced on the 24th that Dr. Chung Cho-rok’s research group at the Stem Cell Research Center has developed a technology of genetic treatment for hereditary spastic paraplegia syndrome (HSP), a rare and incurable disease.
HSP is an inherited neurological disease in which the leg muscles gradually become stiff and weak, leading to paralysis. It occurs in 1.8 out of 100,000 people worldwide.
More than 80 types of genes are known to be intricately involved in the disease, making it difficult to develop a treatment, so we are focusing on alleviating the typical symptoms of lower limb stiffness and muscle loss.
The research team recently discovered the mechanism of development of HSP caused by the “ARL6IP1” gene.
In an experimental disease model in mice, ARL6IP1 was confirmed to exist in the mitochondria-connected endoplasmic reticulum membrane (MAM) and participate in organelle homeostasis to regulate neuronal damage caused by neuroinflammation.
When damaged mitochondria accumulate in nerve cells due to abnormal regulation of autophagy caused by loss of function of ARL6IP1, neurodegeneration occurs and HSP develops.
Based on this mechanism, the research team developed the HSP gene therapy technology and completed the verification of efficacy in an animal model for the first time.
Experimental mice with HSP disease treated with a gene therapy made by loading ARL6IP1 into an adeno-associated virus (AAV) carrier showed decreased lower limb stiffness, improved gait disturbances, and improved brain and brain tissue lesions. of neuroinflammatory responses.
Dr Chung Cho-rok said: “It is significant that it suggests a new mechanism for HSP and opens the possibility of gene therapy.”
The results of this study were published in the latest issue of the ‘Journal of Experimental Medicine’, a leading medical journal.
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01/24/2024 15:49 Sent
2024-01-24 06:49:59
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