Are we creating a new phenotype? Physiological barriers and ethical considerations in the treatment of hereditary transthyretin-amyloidosis

Are we creating a new phenotype? Physiological barriers and ethical considerations in the treatment of hereditary transthyretin-amyloidosis

Abstract Hereditary transthyretin (TTR) amyloidosis (ATTRv) is an autosomal dominant, systemic disease transmitted by amyloidogenic mutations in the TTR gene. To prevent the otherwise fatal disease course, TTR stabilizers and mRNA silencing antisense drugs are currently approved treatment options. W...

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Bibliographic Details
Main Authors: Maike F. Dohrn, Jessica Medina, Karmele R. Olaciregui Dague, Ernst Hund
Format: Article
Language:English
Published: BMC 2021-11-01
Series:Neurological Research and Practice
Subjects:
Central nervous system amyloidosis
Eye involvement
Blood–brain barrier
Liver transplantation
Antisense oligonucleotides
Small interfering RNA
Online Access:https://doi.org/10.1186/s42466-021-00155-8

Internet

https://doi.org/10.1186/s42466-021-00155-8

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