Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

May 20th, 2021|Categories: Perspectives on Current Science|

Imagine if your muscles began weakening for no apparent reason. Then, after an ongoing search for a diagnosis, you learn that you have a disease in which your muscles would progressively waste away and become weaker until you would no longer be able to ...

Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted

April 14th, 2021|Categories: 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 ...

siRNA: From a Powerful Research Tool to Potent Therapeutic Modality

March 30th, 2021|Categories: Perspectives on Current Science|

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 ...

The Astonishing History, Controversies, and Game-Changing Uses of CRISPR

March 16th, 2021|Categories: Perspectives on Current Science|

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 ...

Facts about COVID-19 mRNA Vaccines and the Decades of Research That Went into Creating Them

February 11th, 2021|Categories: Perspectives on Current Science|

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 ...

20th Anniversary of RNA Interference in Mammalian Cells

January 25th, 2021|Categories: Perspectives on Current Science|

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, ...

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