Tag : exon

Latest News

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect

Newsemia
Domenger, C; Allais, M; François, V; Léger, A; Lecomte, E; Montus, M; Servais, L; … Le Guiner, C; + view all Domenger, C; Allais, M;...
Pharma / Biotech

The Pharmacokinetics of 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy.

Newsemia
Related Articles The Pharmacokinetics of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2019 Aug 20;:...
Latest News

New drug provides hope for patients with Duchenne muscular dystrophy

Newsemia
The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy. Source link...
Neurology

High-grade neuroepithelial tumor with BCOR exon 15 internal tandem duplication – a comprehensive clinical, radiographic, pathologic, and genomic analysis.

Newsemia
Related Articles High-grade neuroepithelial tumor with BCOR exon 15 internal tandem duplication – a comprehensive clinical, radiographic, pathologic, and genomic analysis. Brain Pathol. 2019 May...
Pediatrics

Two novel mutations in exon 3 of PHOX2B gene: think about congenital central hypoventilation syndrome in patients with Hirschsprung disease

Newsemia
Congenital central hypoventilation syndrome (CCHS) is characterized by alveolar hypoventilation increasing during sleep and affected patients are unable to perceive and respond to hypercarbia with...
Latest News

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Newsemia
Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have...
Gastroenterology

Capecitabine plus bevacizumab versus capecitabine in maintenance treatment for untreated characterised KRAS exon 2 wild-type metastatic colorectal cancer: a retrospective analysis in Chinese postmenopausal women

Newsemia
Capecitabine plus bevacizumab (CAP-B) maintenance treatment after 6 cycles of capecitabine, oxaliplatin, and bevacizumab (CAPOXB) has demonstrated clinical activity and failure to compromise quality of life...
Latest News

Study points to potential new biomarker and drug target for amyotrophic lateral sclerosis

Newsemia
Research led by stem cell scientists at Harvard University points to a potential new biomarker and drug target for amyotrophic lateral sclerosis, a neurological disease...
Neurology

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy.

Newsemia
NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Sep 27;13:442-449 Authors: Watanabe N,...
Neurology

Urine exRNA may be source of biomarkers for muscular dystrophy

Newsemia
Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular...

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