Elsevier

The Lancet Neurology

Volume 20, Issue 1, January 2021, Pages 21-23
The Lancet Neurology

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Patisiran in hereditary transthyretin-mediated amyloidosis

https://doi.org/10.1016/S1474-4422(20)30397-5Get rights and content

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References (10)

  • SJ Richardson

    Cell and molecular biology of transthyretin and thyroid hormones

    Int Rev Cytol

    (2007)
  • M Luigetti et al.

    Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: current perspectives on improving patient care

    Ther Clin Risk Manag

    (2020)
  • T Coelho et al.

    Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

    J Neurol

    (2013)
  • D Adams et al.

    Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis

    N Engl J Med

    (2018)
  • MD Benson et al.

    Inotersen treatment for patients with hereditary transthyretin amyloidosis

    N Engl J Med

    (2018)
There are more references available in the full text version of this article.

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    Citation Excerpt :

    This LNP is coated by the host apolipoprotein E (ApoE) that triggers its transport into hepatocytes via low-density lipoprotein receptor (LDLR)-mediated endocytosis. The treatment suppresses the deposition of amyloid fibrils and was approved as the first siRNA drug (patisiran) by the FDA in 2018 for the treatment of TTR-type familial amyloid polyneuropathy.61 A similar strategy is under clinical development for the treatment of elevated LDL-cholesterol and hypercholesterolemia using siRNA-LNPs targeting PCSK9 and ApoB, respectively.62

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