Advances in translational research in neuromuscular diseases

Research output: Contribution to journalArticle

Abstract

New therapies developed over the past 3 years for previously intractable diseases of skeletal muscle, neuromuscular junctions, peripheral nerves, and motor neurons are now being incorporated into our standard neuromuscular clinical practice. The past 3 years were also marked by important advances in our understanding of the pathogenesis and pathophysiology of inherited and acquired neuromuscular diseases; these advances were acquired by the use of high-throughput nucleotide and protein analytic methods, novel animal models, and human-induced pluripotent stem cell-derived "diseases in a dish." Over the next decade, we can reasonably anticipate that these insights, coupled with advances in our ability to modulate immune mechanisms, to modify the activity of mutant genes, and to perform gene replacement therapies with enhanced viral vector-based and stem cell-based delivery systems, will revolutionize our management of neuromuscular diseases.

Original languageEnglish (US)
Pages (from-to)429-433
Number of pages5
JournalArchives of Neurology
Volume68
Issue number4
DOIs
StatePublished - Apr 2011

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Neuromuscular Diseases
Translational Medical Research
Induced Pluripotent Stem Cells
Neuromuscular Junction
Motor Neurons
Peripheral Nerves
Genetic Therapy
Skeletal Muscle
Stem Cells
Nucleotides
Animal Models
Genes
Proteins
Neuromuscular Disease
Therapy
Gene
Translational Research
Therapeutics
Clinical Practice
Protein

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Advances in translational research in neuromuscular diseases. / Pleasure, David E.

In: Archives of Neurology, Vol. 68, No. 4, 04.2011, p. 429-433.

Research output: Contribution to journalArticle

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