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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共 202 条
  • [91] Hutchinson J, 1886, Med Chir Trans, V69, P473
  • [92] B-type lamins in health and disease
    Hutchison, C. J.
    [J]. SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY, 2014, 29 : 158 - 163
  • [93] Targeting Isoprenylcysteine Methylation Ameliorates Disease in a Mouse Model of Progeria
    Ibrahim, Mohamed X.
    Sayin, Volkan I.
    Akula, Murali K.
    Liu, Meng
    Fong, Loren G.
    Young, Stephen G.
    Bergo, Martin O.
    [J]. SCIENCE, 2013, 340 (6138) : 1330 - 1333
  • [94] A truncated lamin A in the Lmna-/- mouse line Implications for the understanding of laminopathies
    Jahn, Daniel
    Schramm, Sabine
    Schnoelzer, Martina
    Heilmann, Clemens J.
    de Koster, Chris G.
    Schuetz, Wolfgang
    Benavente, Ricardo
    Alsheimer, Manfred
    [J]. NUCLEUS-AUSTIN, 2012, 3 (05): : 463 - 474
  • [95] New Lmna knock-in mice provide a molecular mechanism for the 'segmental aging' in Hutchinson-Gilford progeria syndrome
    Jung, Hea-Jin
    Tu, Yiping
    Yang, Shao H.
    Tatar, Angelica
    Nobumori, Chika
    Wu, Daniel
    Young, Stephen G.
    Fong, Loren G.
    [J]. HUMAN MOLECULAR GENETICS, 2014, 23 (06) : 1506 - 1515
  • [96] Regulation of prelamin A but not lamin C by miR-9, a brain-specific microRNA
    Jung, Hea-Jin
    Coffinier, Catherine
    Choe, Youngshik
    Beigneux, Anne P.
    Davies, Brandon S. J.
    Yang, Shao H.
    Barnes, Richard H., II
    Hong, Janet
    Sun, Tao
    Pleasure, Samuel J.
    Young, Stephen G.
    Fong, Loren G.
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2012, 109 (07) : E423 - E431
  • [97] Chemical screening identifies ROCK as a target for recovering mitochondrial function in Hutchinson-Gilford progeria syndrome
    Kang, Hyun Tae
    Park, Joon Tae
    Choi, Kobong
    Choi, Hyo Jei Claudia
    Jung, Chul Won
    Kim, Gyu Ree
    Lee, Young-Sam
    Park, Sang Chul
    [J]. AGING CELL, 2017, 16 (03): : 541 - 550
  • [98] Kang SM, 2021, COMMUN BIOL, V4, DOI 10.1038/s42003-020-01540-w
  • [99] Rapamycin Rescues Age-Related Changes in Muscle-Derived Stem/Progenitor Cells from Progeroid Mice
    Kawakami, Yohei
    Hambright, William S.
    Takayama, Koji
    Mu, Xiaodong
    Lu, Aiping
    Cummins, James H.
    Matsumoto, Tomoyuki
    Yurube, Takashi
    Kuroda, Ryosuke
    Kurosaka, Masahiro
    Fu, Freddie H.
    Robbins, Paul D.
    Niedernhofer, Laura J.
    Huard, Johnny
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019, 14 : 64 - 76
  • [100] In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice
    Koblan, Luke W.
    Erdos, Michael R.
    Wilson, Christopher
    Cabral, Wayne A.
    Levy, Jonathan M.
    Xiong, Zheng-Mei
    Tavarez, Urraca L.
    Davison, Lindsay M.
    Gete, Yantenew G.
    Mao, Xiaojing
    Newby, Gregory A.
    Doherty, Sean P.
    Narisu, Narisu
    Sheng, Quanhu
    Krilow, Chad
    Lin, Charles Y.
    Gordon, Leslie B.
    Cao, Kan
    Collins, Francis S.
    Brown, Jonathan D.
    Liu, David R.
    [J]. NATURE, 2021, 589 (7843) : 608 - +