Preclinical Advances of Therapies for Laminopathies

作者：Louise Benarroch, Enzo Cohen, António Atalaia, Rabah Ben Yaou, Gisèle Bonne, A. Bertrand · 发表于：Journal of Clinical Medicine · 年份：2021 · DOI：10.3390/jcm10214834 · 被引用次数：14 · 研究领域：Nuclear Structure and Function、RNA Research and Splicing、RNA regulation and disease

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.