Perspectives on Current Science

Recently, people who have been impacted by Huntington’s disease, their families, and the OTS community were disappointed to learn that the GENERATION HD1 Phase III study of Tominersen was halted based on the recommendation of an unblinded Independent Data Monitoring Committee. Although not based ...

Earlier this year we celebrated the 20th Anniversary of RNA Interference in Mammalian Cells. In this article, we are going to discuss small interfering RNA (siRNA), a key mechanism that induces gene silencing in the RNAi pathway. siRNAs are highly specific, making them a ...

Many of us view CRISPR as an incredibly useful tool. It is even referred to as genetic scissors because CRISPR allows scientists to edit the genes (the DNA) of organisms, from the smallest cells to an entire mammal. Because of its revolutionary impact on ...

Imagine if a medication or therapy could be created to treat just one individual with an ultra-rare disease. There are nearly 7,000 known rare diseases (1) and many are debilitating and ultimately fatal. In the U.S., a disease is considered rare when it affects ...

Posted: February 11, 2021 | Updated: October 8, 2021 Despite the global threat of COVID-19, mRNA vaccines, which are highly effective at preventing COVID-19 illness and have passed rigorous safety standards, have been met with skepticism by many people. This suspicion is due in ...

The groundbreaking discovery of RNA interference by Craig Mello and Andrew Fire was swiftly followed by the discovery of RNAi as a gene-silencing mechanism in mammalian cells. Since these breakthroughs, members of our community have devoted their lives to building on these exciting discoveries, ...

Oligonucleotide therapies are relatively new in the field of medicine, but they have allowed us to make astounding progress in the fight against COVID-19. Rather than simply repurposing existing drugs or spending years to develop new drugs and vaccines, oligo therapies are quickly providing ...

In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising technologies to fight the coronavirus are based on a new type of drug called oligonucleotide ...