NEW YORK (GenomeWeb) – Dicerna Pharmaceuticals this week announced preclinical data showing that its investigational primary hyperoxaluria type 1 (PH1) therapy DCR-PH1 could silence expression of its target by as much as 97 percent in a mouse model of the disease.

PH1 is a rare, inherited autosomal-recessive condition characterized by the liver's inability to metabolize a precursor of oxalate due to disruption of an enzyme called alanine-glyoxylate aminotransferase 1 (AGT1). As a result, calcium oxalate builds up in renal tubules causing kidney stones and fibrosis.

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Sophia Genetics

With the Next Generation Sequencing (NGS), genomes sequencing has been democratized over the last decades with the detection of genomic alterations, thus replacing Sanger sequencing.

Nov
15
Sponsored by
Twist Bioscience

This webinar will discuss how Amyris, a biotechnology company that develops renewable products for a broad range of applications and industries, uses large-scale microbial engineering to support its manufacturing processes.