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Preclinical Advances of Therapies for Laminopathies

Laminopathies are a group of rare disorders due to mutation in LMNA gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fu...

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Autores principales: Benarroch, Louise, Cohen, Enzo, Atalaia, Antonio, Ben Yaou, Rabah, Bonne, Gisèle, Bertrand, Anne T
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8584472/
https://www.ncbi.nlm.nih.gov/pubmed/34768351
http://dx.doi.org/10.3390/jcm10214834
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author Benarroch, Louise
Cohen, Enzo
Atalaia, Antonio
Ben Yaou, Rabah
Bonne, Gisèle
Bertrand, Anne T
author_facet Benarroch, Louise
Cohen, Enzo
Atalaia, Antonio
Ben Yaou, Rabah
Bonne, Gisèle
Bertrand, Anne T
author_sort Benarroch, Louise
collection PubMed
description Laminopathies are a group of rare disorders due to mutation in LMNA gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes.
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spelling pubmed-85844722021-11-12 Preclinical Advances of Therapies for Laminopathies Benarroch, Louise Cohen, Enzo Atalaia, Antonio Ben Yaou, Rabah Bonne, Gisèle Bertrand, Anne T J Clin Med Review Laminopathies are a group of rare disorders due to mutation in LMNA gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes. MDPI 2021-10-21 /pmc/articles/PMC8584472/ /pubmed/34768351 http://dx.doi.org/10.3390/jcm10214834 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Benarroch, Louise
Cohen, Enzo
Atalaia, Antonio
Ben Yaou, Rabah
Bonne, Gisèle
Bertrand, Anne T
Preclinical Advances of Therapies for Laminopathies
title Preclinical Advances of Therapies for Laminopathies
title_full Preclinical Advances of Therapies for Laminopathies
title_fullStr Preclinical Advances of Therapies for Laminopathies
title_full_unstemmed Preclinical Advances of Therapies for Laminopathies
title_short Preclinical Advances of Therapies for Laminopathies
title_sort preclinical advances of therapies for laminopathies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8584472/
https://www.ncbi.nlm.nih.gov/pubmed/34768351
http://dx.doi.org/10.3390/jcm10214834
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