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

来源：

JOURNAL OF CLINICAL MEDICINE | 2021年 / 10卷 / 21期

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.

引用

页数：30

共 202 条

[31] A farnesyltransferase inhibitor prevents both the onset and late progression of cardiovascular disease in a progeria mouse model [J].

Capell, Brian C. ;

Olive, Michelle ;

Erdos, Michael R. ;

Cao, Kan ;

Faddah, Dina A. ;

Tavarez, Urraca L. ;

Conneely, Karen N. ;

Qu, Xuan ;

San, Hong ;

Ganesh, Santhi K. ;

Chen, Xiaoyan ;

Avallone, Hedwig ;

Kolodgie, Frank D. ;

Virmani, Renu ;

Nabel, Elizabeth G. ;

Collins, Francis S. .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2008, 105 (41) :15902-15907

[32] Myoblasts and Embryonic Stem Cells Differentially Engraft in a Mouse Model of Genetic Dilated Cardiomyopathy [J].

Catelain, Cyril ;

Riveron, Stephanie ;

Papadopoulos, Aurelie ;

Mougenot, Nathalie ;

Jacquet, Adeline ;

Vauchez, Karine ;

Yada, Erica ;

Puceat, Michel ;

Fiszman, Marc ;

Butler-Browne, Gillian ;

Bonne, Gisele ;

Vilquin, Jean-Thomas .

MOLECULAR THERAPY, 2013, 21 (05) :1064-1075

[33] Heterozygous LmnadelK32 mice develop dilated cardiomyopathy through a combined pathomechanism of haploinsufficiency and peptide toxicity [J].

Cattin, Marie-Elodie ;

Bertrand, Anne T. ;

Schlossarek, Saskia ;

Le Bihan, Marie-Catherine ;

Skov Jensen, Soren ;

Neuber, Christiane ;

Crocini, Claudia ;

Maron, Sophia ;

Laine, Jeanne ;

Mougenot, Nathalie ;

Varnous, Shaida ;

Fromes, Yves ;

Hansen, Arne ;

Eschenhagen, Thomas ;

Decostre, Valerie ;

Carrier, Lucie ;

Bonne, Gisele .

HUMAN MOLECULAR GENETICS, 2013, 22 (15) :3152-3164

[34] Autophagic degradation of farnesylated prelamin A as a therapeutic approach to lamin-linked progeria [J].

Cenni, V. ;

Capanni, C. ;

Columbaro, M. ;

Ortolani, M. ;

D'Apice, M. R. ;

Novelli, G. ;

Fini, M. ;

Marmiroli, S. ;

Scarano, E. ;

Maraldi, N. M. ;

Squarzoni, S. ;

Prencipe, S. ;

Lattanzi, G. .

EUROPEAN JOURNAL OF HISTOCHEMISTRY, 2011, 55 (04) :200-205

[35] Rapamycin treatment of Mandibuloacral Dysplasia cells rescues localization of chromatin-associated proteins and cell cycle dynamics [J].

Cenni, Vittoria ;

Capanni, Cristina ;

Mattioli, Elisabetta ;

Columbaro, Marta ;

Wehnert, Manfred ;

Ortolani, Michela ;

Fini, Milena ;

Novelli, Giuseppe ;

Bertacchini, Jessika ;

Maraldi, Nadir M. ;

Marmiroli, Sandra ;

D'Apice, Maria Rosaria ;

Prencipe, Sabino ;

Squarzoni, Stefano ;

Lattanzi, Giovanna .

AGING-US, 2014, 6 (09) :755-+

[36] The phenotypic manifestations of autosomal recessive axonal Charcot-Marie-Tooth due to a mutation in Lamin A/C gene [J].

Chaouch, M ;

Allal, Y ;

De Sandre-Giovannoli, A ;

Vallat, JM ;

Amer-el-Khedoud, A ;

Kassouri, N ;

Chaouch, A ;

Sindou, P ;

Hammadouche, T ;

Tazir, M ;

Lévy, N ;

Grid, D .

NEUROMUSCULAR DISORDERS, 2003, 13 (01) :60-67

[37] Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation [J].

Chatzifrangkeskou, Maria ;

Yadin, David ;

Marais, Thibaut ;

Chardonnet, Solenne ;

Cohen-Tannoudji, Mathilde ;

Mougenot, Nathalie ;

Schmitt, Alain ;

Crasto, Silvia ;

Di Pasquale, Elisa ;

Macquart, Coline ;

Tanguy, Yannick ;

Jebeniani, Imen ;

Puceat, Michel ;

Rodriguez, Blanca Morales ;

Goldmann, Wolfgang H. ;

Dal Ferro, Matteo ;

Biferi, Maria-Grazia ;

Knaus, Petra ;

Bonne, Gisele ;

Worman, Howard J. ;

Muchir, Antoine .

HUMAN MOLECULAR GENETICS, 2018, 27 (17) :3060-3078

[38] ERK1/2 directly acts on CTGF/CCN2 expression to mediate myocardial fibrosis in cardiomyopathy caused by mutations in the lamin A/C gene [J].

Chatzifrangkeskou, Maria ;

Le Dour, Caroline ;

Wu, Wei ;

Morrow, John P. ;

Joseph, Leroy C. ;

Beuvin, Maud ;

Sera, Fusako ;

Homma, Shunichi ;

Vignier, Nicolas ;

Mougenot, Nathalie ;

Bonne, Gisele ;

Lipson, Kenneth E. ;

Worman, Howard J. ;

Muchir, Antoine .

HUMAN MOLECULAR GENETICS, 2016, 25 (11) :2220-2233

[39] Genomic Reorganization of Lamin-Associated Domains in Cardiac Myocytes Is Associated With Differential Gene Expression and DNA Methylation in Human Dilated Cardiomyopathy [J].

Cheedipudi, Sirisha M. ;

Matkovich, Scot J. ;

Coarfa, Cristian ;

Hu, Xin ;

Robertson, Matthew J. ;

Sweet, Mary ;

Taylor, Matthew ;

Mestroni, Luisa ;

Cleveland, Joseph ;

Willerson, James T. ;

Gurha, Priyatansh ;

Marian, Ali J. .

CIRCULATION RESEARCH, 2019, 124 (08) :1198-1213

[40] LMNA mutations in atypical Werner's syndrome [J].

Chen, LS ;

Lee, L ;

Kudlow, BA ;

Dos Santos, HG ;

Sletvold, O ;

Shafeghati, Y ;

Botha, EG ;

Garg, A ;

Hanson, NB ;

Martin, GM ;

Mian, IS ;

Kennedy, BK ;

Oshima, J .

LANCET, 2003, 362 (9382) :440-445

← 1 2 3 4 5 6 7 8 9 10 →