echemi logo
Product
  • Product
  • Supplier
  • Inquiry
    Home > Biochemistry News > Biotechnology News > "Nature Medicine" Silencing a gene accidentally found clues to the treatment of ALS

    "Nature Medicine" Silencing a gene accidentally found clues to the treatment of ALS

    • Last Update: 2022-02-18
    • Source: Internet
    • Author: User
    Search more information of high quality chemicals, good prices and reliable suppliers, visit www.echemi.com

    NIH-funded preclinical study identifies potential treatment for ALS

    Using an experimental drug, researchers were able to suppress a mutant amyotrophic lateral sclerosis (ALS) gene


    Dr.


    ALS is a fatal neurological disease that causes motor neurons in the brain and spinal cord to degenerate


    In this study, an antisense oligonucleotide drug was used to inhibit FUS by preventing cells from producing a specific protein, thereby delaying the onset of motor neuron degeneration in mice


    Compared with normal mice, mice with mutations in the FUS gene had higher levels of insoluble FUS and other ALS-related proteins in the brain and spinal cord


    This study establishes a highly disease-related mouse model where FUS toxicity is due to increased function and is dose-dependent, suggesting that we can treat FUS-ALS by silencing the FUS gene


    In 2019, author Dr.


    In mice, a single dose of the drug injected into the ventricle, the fluid-filled space around the brain, delayed the onset of inflammation and motor neuron degeneration by six months


    Dr.


    Dr Schneider said: "This study is an example of precision medicine, a bed-to-bed effort


    Treatment began more than 6 months after clinical onset, by which time the disease had significantly worsened


    By studying patients' brain and spinal cord tissue, the researchers found that the drug silenced FUS throughout the nervous system and reversed the toxicity of FUS and other disease-related proteins


    FUS has been shown to be important in various cellular processes


    Korobeynikov, VA, et al.



    This article is an English version of an article which is originally in the Chinese language on echemi.com and is provided for information purposes only. This website makes no representation or warranty of any kind, either expressed or implied, as to the accuracy, completeness ownership or reliability of the article or any translations thereof. If you have any concerns or complaints relating to the article, please send an email, providing a detailed description of the concern or complaint, to service@echemi.com. A staff member will contact you within 5 working days. Once verified, infringing content will be removed immediately.

    Contact Us

    The source of this page with content of products and services is from Internet, which doesn't represent ECHEMI's opinion. If you have any queries, please write to service@echemi.com. It will be replied within 5 days.

    Moreover, if you find any instances of plagiarism from the page, please send email to service@echemi.com with relevant evidence.