Are we close to targeted treatments for Huntington’s disease? YES

Authors: prof. MUDr. Jan Roth, CSc.
Authors‘ workplace: 1. LF UK a VFN v Praze ;  Centrum extrapyramidových, onemocnění, Neurologická klinika, a Centrum klinických neurověd
Published in: Cesk Slov Neurol N 2020; 83/116(3): 240
Category: Controversions

  1. Rodriguesa FB, Quinn L, Wild EJ. Huntington’s disease clinical trials. corner: january 2019. J Hunting Dis 2019; 8: 115–125.

  2. Smith AV, Tabrizi SJ. Therapeutic Antisense targeting of huntingtin. DNA Cell Biol 2020; 39(2): 154–158.

  3. Scoles DR, Minikel EV, Pulst SM. Antisense oligonucleotides: a primer. Neurol Genet 20191; 5(2): e323.

  4. Singh KRoy I. Nucleic acid therapeutics in Huntington's disease. Recent Pat Biotechnol 2019; 13(3): 187–206.

  5. Su Y, Renbao C, Huiming Y et al. CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease. J Clin Invest 2017; 127(7): 2719–2724.

  6. Tabrizi SJ, Leavitt BR, Landwehrmeyer GB et al. Phase 1–2a IONIS-HTTRx study site teams. Targeting Huntingtin Expression in patients with huntington's disease. N Engl J Med 2019; 380(24): 2307–2316.

  7. Zain R, Smith CI. targeted oligonucleotides for treating neurodegenerative tandem repeat diseases. Neurotherapeutics 2019; 16(2): 248–262.

Paediatric neurology Neurosurgery Neurology

Article was published in

Czech and Slovak Neurology and Neurosurgery

Issue 3

2020 Issue 3

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