New and Upcoming Research in Therapeutics - Jee Bang and Wenzhen Duan

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  • เผยแพร่เมื่อ 10 ม.ค. 2022
  • (November 6, 2021) Jee Bang, MD, MPH, and Wenzhen Duan, MD, Ph.D. from Johns Hopkins University gives a review on current therapies in HD clinical trials and outlines new therapeutic approaches.
    It is an exciting time for CRISPR gene editing therapy. Dr. Duan speaks on HD gene targeting therapies. CRISPR/Cas9-medicated non-allele selective HTT-lowering at the premanifest stage delays the onset and slows disease progression in HD preclinical studies, Allele-selective mutant HTT-lowering approaches are promising for further development, Lipid nanoparticle-formulation has the potential to effectively and noninvasively deliver gene therapies to the brain.

ความคิดเห็น • 1

  • @sismarin
    @sismarin 7 หลายเดือนก่อน

    Are there no studies with methylene blue?