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

ABSTRACT:

Background

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.

Methods

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 angi

SUBMITTER: Loboda A 

PROVIDER: S-EPMC7550322 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature