Preclinical Advances of Therapies for Laminopathies

被引:9
作者
Benarroch, Louise [1 ]
Cohen, Enzo [1 ]
Atalaia, Antonio [1 ]
Ben Yaou, Rabah [1 ]
Bonne, Gisele [1 ]
Bertrand, Anne T. [1 ]
机构
[1] Sorbonne Univ, INSERM, Inst Myol, Ctr Rech Myol, F-75013 Paris, France
关键词
LMNA; lamin A/C; progerin; laminopathy; treatment; therapy; HGPS; preclinical models; GILFORD PROGERIA SYNDROME; LAMIN A/C GENE; CHARCOT-MARIE-TOOTH; DREIFUSS MUSCULAR-DYSTROPHY; LMNA-MUTATIONS CAUSE; STEM-CELL THERAPY; MOUSE MODEL; DILATED CARDIOMYOPATHY; FARNESYLTRANSFERASE INHIBITOR; NUCLEAR-ENVELOPE;
D O I
10.3390/jcm10214834
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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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页数:30
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