Cargando…

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...

Descripción completa

Detalles Bibliográficos
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
Descripción
Sumario: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.