Traiter les neuropathies héréditaires : un rêve qui devient réalité ? [Treating hereditary neuropathies : a dream come true?]

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State: Public
Version: Final published version
License: CC BY-NC-ND 4.0
Serval ID
serval:BIB_AB887B515538
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Traiter les neuropathies héréditaires : un rêve qui devient réalité ? [Treating hereditary neuropathies : a dream come true?]
Journal
Revue medicale suisse
Author(s)
Echaniz-Laguna A., Magy L., Vicino A., Fayolle D., Hübers A., Ochsner F., Théaudin M.
ISSN
1660-9379 (Print)
ISSN-L
1660-9379
Publication state
Published
Issued date
27/04/2022
Peer-reviewed
Oui
Volume
18
Number
779
Pages
813-816
Language
french
Notes
Publication types: Journal Article
Publication Status: ppublish
Abstract
Hereditary neuropathies have been the subject of recent major therapeutic advances. Treatments based on antisense oligonucleotides (ASO) and small interfering RNA (siRNA) have been developed and are now commercially available to treat hereditary transthyretin amyloidosis (hTTR) and porphyria. More recently, a CRISPR-Cas9 genomic editing treatment targeting the TTR gene has been developed and is being tested in patients with hTTR. Based on their success in hTTR and porphyria, innovative treatments targeting mRNA and DNA are being evaluated in other hereditary neuropathies, including Charcot-Marie-Tooth disease (CMT).
Keywords
Amyloid Neuropathies, Familial/drug therapy, Amyloid Neuropathies, Familial/therapy, Charcot-Marie-Tooth Disease/genetics, Charcot-Marie-Tooth Disease/therapy, Humans
Pubmed
Create date
16/05/2022 10:54
Last modification date
17/08/2022 7:13
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