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 walk. Ultimately, the disease would lead to death. This is exactly what people diagnosed with ...
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 diseases? What about brain tumors and cancers, Alzheimer’s disease, Multiple Sclerosis, stroke, or behavioral conditions? ...
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 on emergent safety concerns, the specific reasoning behind this recommendation has not yet been released. ...
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 powerful tool to suppress the expression of specific genes, but they did require a significant ...
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 life sciences, the discovery of CRISPR as a genome editor was awarded a Nobel Prize ...
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 as many as 200,000 people. In that situation, it is difficult enough to find an ...
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 ...
Can siRNA Therapeutics Heal the Brain?
As we’ve reviewed in past articles the discovery and history of RNA interference and siRNA ...
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 ...
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 ...
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 ...
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 ...
