Systematic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Article Abstract:

The role of antisense therapy in treating individuals with Duchenne muscular dystrophy (DMD) is examined. The expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse is induced by the weekly intravenous injections of morpholino phosphorordiamidate (morpholino) antisense oligonucleotide (AON) thereby improving muscle function.

United States, Care and treatment, Drug therapy, Dosage and administration, Antisense drugs, Duchenne muscular dystrophy

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A critical developmental switch defines the kinetics of kidney cyst formation after loss of Pkd1

Article Abstract:

The mutations in PKD1 (the gene encoding polycystin-1) are the main cause of autosomal dominant polycystic kidney disease, which is an important cause of end-stage renal disease. Findings show that Pkd1 has regulated tubular morphology in both developing and adult kidney, but the pathologic consequences of inactivation are defined by the developmental status of the organ.

Genetic aspects, Technology application, Fluorescence, Gene expression, Polycystic kidney disease

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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

Article Abstract:

The majority of mutations in the dystrophin gene occur in the region encoding the spectrin like central rod domain, which is largely dispensable. Tests are conducted in vivo in the mdx dystrophic mouse by combining a potent transfection protocol with a 2-o-methylated phosphorothioated antisense oligoribonucleotide designed to promote skipping of the mutated exon.

Exon (Molecular genetics), Exons (Molecular genetics), Dystrophin

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