A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared interim data from the first ever human study using systemically administered CRISPR as a gene editing therapy. Early results that surpassed expectations show that not only is the CRISPR-based ...
Oligonucleotide Therapeutics – Restoring Sight to the Blind
Vision is one of our most priceless senses. Without it, we miss out on precious visuals, such as witnessing a child’s face light up with joy or seeing a glorious sunset, and even the important relational activity of reading the expressions on someone’s face ...
Can Oligonucleotides Target RNA Splicing to Treat Disease?
The human body is phenomenally complex, and we are continually discovering incredible facts about it. One fascinating aspect lies in splicing of RNA as it is processed in the cell. Alternate splicing of a single pre-mRNA molecule can create tens of thousands of different ...
mRNA Is Capable of Providing Solutions Far Beyond Vaccines
When the FDA issued emergency use authorization for Pfizer-BioNTech and Moderna’s COVID-19 vaccines, mRNA instantly became a household word. Many people believed that this new technology had sprung up out of nowhere in less than a year. What they did not know is that ...
Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy
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 ...
Can siRNA Therapeutics Heal the Brain?
As we’ve reviewed in past articles the discovery and history of RNA interference and siRNA as a potent therapeutic modality, it brings us to a fascinating idea. Does siRNA carry the potential to heal the brain? If so, can it treat more than genetic ...
Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted
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
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
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 ...
The Emerging Era of N-of-1 Drugs for Ultra-Rare Genetic Diseases
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 ...
