Feng et al. (2015) recently reviewed the progress in nanopore technology from the past as well as the latest advances. They describe the different types of nanopores and discuss recent and potential applications. Nanopore-based sequencers have the potential to quickly and reliably sequence the entire human genome “for less than $1000, and possibly for even […]
Posts Categorized: In the news
Short interfering RNAs (siRNAs) are RNA strands designed to bind to target strands of mRNA and knock down the expression of targeted genes, thus preventing the synthesis of the respective proteins. Special interest in recent years about using siRNAs to target key disease proteins has been suppressed due to the difficulty of having the siRNAs […]
Nathwani et al, report in the last issue of NEJM the results of a clinical trial consisting of an Adenovirus-Associated Virus (AAV) vectorâ€“mediated gene transfer in Hemophilia B. The cohort consisted of 6 patients who received low, intermediate or high doses of the vector. AAV-mediated expression of Factor IX (FIX) at 2 to 11% of […]
Genetic Alliance is a nonprofit health advocacy organization that improves health through the authentic engagement of communities and individuals. This year, they are celebrating their 25th anniversary as a journey toward novel partnerships, connected consumers, and smart services. They are hosting a variety of events throughout the year, including monthly salons around the country and […]
Pompe disease is a rare genetic disorder caused by a deficiency of an enzyme – acid alpha-glucosidase (GAA) – which is needed to break down glycogen.