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Preclinical Advances of Therapies for Laminopathies.


ABSTRACT: 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.

SUBMITTER: Benarroch L 

PROVIDER: S-EPMC8584472 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature

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Preclinical Advances of Therapies for Laminopathies.

Benarroch Louise L   Cohen Enzo E   Atalaia Antonio A   Ben Yaou Rabah R   Bonne Gisèle G   Bertrand Anne T AT  

Journal of clinical medicine 20211021 21


Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> 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 the  ...[more]

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