Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy.

Development of Antisense Gapmers for the Treatment of Huntington's Disease.

Development and Clinical Applications of Antisense Oligonucleotide Gapmers.

Development of Antisense Oligonucleotide Gapmers for Amyotrophic Lateral Sclerosis.

Detection of Locked Nucleic Acid and 2'-O-Methoxyethyl Gapmers from Mouse Samples Using ELISA.

A sensitive enzyme-linked immunosorbent assay (ELISA)-based protocol is described that can be used to quantify the levels of locked nucleic acid (LNA) and 2'-O-methoxyethyl (2'-MOE) gapmers in mouse muscle tissue.

Investigation of the pentamidine efficacy against myotonic dystrophy type 1 exploiting polymeric- and lipid-based nanoparticles.

Myotonic dystrophy type 1 (DM1) is a genetic disorder caused by a mutation in the DMPK gene, in which an expanded CTG trinucleotide is transcribed into abnormal mRNA that accumulates in nuclear foci. These foci sequester crucial splicing factors, causing a strong mis-regulation of downstream target genes. Consequently,...

Antisense Oligonucleotide-Mediated Degradation of Toxic RNA in Myotonic Dystrophy Type 1.