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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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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. |
format | Online Article Text |
id | pubmed-8584472 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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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