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Article

Efficiency of silencing RNA for removal of transthyretin V30M in a TTR leptomeningeal animal model.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis - 1 Dec 2016

Gonçalves Paula, Martins Helena, Costelha Susete, Maia Luis F, Saraiva Maria Joao

Abstract excerpt

Some TTR mutants target the central nervous system (CNS). Familial amyloid polyneuropathy (FAP) with leptomeningeal involvement has been described in 9% of transthyretin (TTR) mutations and in valine for methionine at position 30 (V30M) patients. These individuals present dementia, ataxia, brain hemorrhages and focal neurological episodes (FNEs). FNEs occurred also in V30M FAP patients with longer disease...

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Source

Identifiers

PMID: 27884058
DOI: 10.1080/13506129.2016.1256282

Genes

TTR
FAP

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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