Non-Profits Create Next-Generation Medicines that Treat Ultra-Rare Diseases
Hundreds of millions of people worldwide are living with a rare disease. Most rare diseases do not currently have a cure. So, once diagnosed with a rare disease, most patients and their families are merely given strategies to manage the disease symptoms and try ...
Oligonucleotides Safely Cross the Blood-Brain Barrier to Knock Down Gene Expression
Neurodegenerative diseases, brain tumors, and infectious diseases and inflammatory conditions that impact the brain wreak havoc on a person’s health and are notoriously difficult to treat. Oligonucleotide drugs could provide solutions to many pathological brain conditions if certain challenges could be overcome. Researchers at ...
OTS President’s Paper Pick – August 2021
By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian D Gillmore, Ed Gane, Jorg Taube, Justin Kao, Marianna Fontana, Michael L Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, Adam ...
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines and the recent news of phenomenal interim results for Intellia’s investigational genome editing treatment for ATTR amyloidosis. Neither of these would have been possible without a lesser-known component – ...
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 therapy ...
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 ...
