Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.

Pharmacological Reports : PR
Agnieszka Łoboda, Józef Dulak

Abstract

Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular childhood disorder that causes progressive muscle weakness and degeneration and results in functional decline, loss of ambulation and early death of young men due to cardiac or respiratory failure. Although the major cause of the disease has been known for many years-namely mutation in the DMD gene encoding dystrophin, one of the largest human genes-DMD is still incurable, and its treatment is challenging. A comprehensive and systematic review of literature on the gene, cell, and pharmacological experimental therapies aimed at restoring functional dystrophin or to counteract the associated processes contributing to disease progression like inflammation, fibrosis, calcium signaling or angiogenesis was carried out. Although some therapies lead to satisfying effects in skeletal muscle, they are highly ineffective in the heart; therefore, targeting defective cardiac and respiratory systems is vital in DMD patients. Unfortunately, most of the pharmacological compounds treat only the symptoms of the disease. Some drugs addressing the underlying cause, like eteplirsen, golodirsen, and ataluren, have recently been conditionally approved; however, they can correct only...Continue Reading

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Citations

Jan 6, 2021·International Journal of Molecular Sciences·Moises Rodriguez-GonzalezAna Castellano-Martinez
Feb 26, 2021·Pflügers Archiv : European journal of physiology·Kalina AndrysiakJózef Dulak
Mar 7, 2021·Journal of Clinical Medicine·Fernanda FortunatoAlessandra Ferlini
Apr 8, 2021·Cellular and Molecular Life Sciences : CMLS·Alicja Starosta, Patryk Konieczny
Jun 7, 2021·Cellular and Molecular Life Sciences : CMLS·Urszula Florczyk-SoluchJózef Dulak
Jun 5, 2021·Frontiers in Immunology·Bradley A Hamilton, J Fraser Wright
Jul 14, 2021·Cardiovascular Research·Francesco CanonicoDomenico D'Amario
Aug 8, 2021·International Journal of Molecular Sciences·Shalini Murali KrishnanPeter Sandner
Aug 20, 2021·Frontiers in Genetics·Olivier BoyerUNKNOWN Study Group

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Methods Mentioned

BETA
antisense oligonucleotides
biopsy
antisense oligonucleotide
chemical modifications
glycosylation
genetic modification
transfection

Clinical Trials Mentioned

NCT03375164
NCT03769116
NCT03362502
NCT03368742
NCT02420379
NCT02530905
NCT02310906
NCT02500381
NCT04179409
NCT03532542

Software Mentioned

Solid
ReveraGen

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