Charcot-Marie-Tooth (CMT) neuropathies are one of the most common neuromuscular disorders. However, despite the identification of more than 100 causative genes, therapeutic options are still missing. The generation of authentic animal models and the increasing insights into the understanding of disease mechanisms, in addition to extraordinary developments in gene and molecular therapies, are quickly changing this scenario, and several strategies are currently being translated, or are getting close to, clinical trials. Here, we provide an overview of the most recent advances for the therapy of CMT at both the preclinical and clinical levels. For clarity, we have grouped the approaches in three different categories: gene therapy based on viral-mediated delivery, molecular therapies based on alternative delivery systems, and pharmacological therapies.

Recent advances in the treatment of Charcot-Marie-Tooth neuropathies / Bolino, Alessandra; D'Antonio, Maurizio. - In: JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM. - ISSN 1085-9489. - 28:2(2023), pp. 1-16. [10.1111/jns.12539]

Recent advances in the treatment of Charcot-Marie-Tooth neuropathies

Alessandra Bolino
Primo
;
2023-01-01

Abstract

Charcot-Marie-Tooth (CMT) neuropathies are one of the most common neuromuscular disorders. However, despite the identification of more than 100 causative genes, therapeutic options are still missing. The generation of authentic animal models and the increasing insights into the understanding of disease mechanisms, in addition to extraordinary developments in gene and molecular therapies, are quickly changing this scenario, and several strategies are currently being translated, or are getting close to, clinical trials. Here, we provide an overview of the most recent advances for the therapy of CMT at both the preclinical and clinical levels. For clarity, we have grouped the approaches in three different categories: gene therapy based on viral-mediated delivery, molecular therapies based on alternative delivery systems, and pharmacological therapies.
2023
Charcot-Marie-Tooth, therapy
File in questo prodotto:
File Dimensione Formato  
J Peripheral Nervous Sys - 2023 - Bolino - Recent advances in the treatment of Charcot‐Marie‐Tooth neuropathies.pdf

accesso aperto

Tipologia: PDF editoriale (versione pubblicata dall'editore)
Licenza: Creative commons
Dimensione 1.02 MB
Formato Adobe PDF
1.02 MB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11768/140016
Citazioni
  • ???jsp.display-item.citation.pmc??? 13
  • Scopus 17
  • ???jsp.display-item.citation.isi??? 15
social impact