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 P Boyd, Jeffrey E Cehelsky, Mark D McKee, Andrew Schiermeier, Olivier Harari, Andrew Murphy, Christos ...
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 – lipid nanoparticles. This delivery system has been decades in the making and, fortunately for humanity, ...
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 to treat ATTR amyloidosis safe, it also precisely edited the desired gene in the ...
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 during a conversation. Some people are born without the gift of sight and others endure ...
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 mRNAs, which contributes to the existence of such an intricate organism as a human. If ...
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 it had been in development for two decades, for uses ranging from vaccines for infectious ...
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 ...
Lipid Nanoparticles: Nanomedicine’s Triumph
Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines ...
A Momentous CRISPR Milestone was Achieved with Remarkable Results
Intellia Therapeutics and Regeneron Pharmaceuticals’ recent announcement is a major milestone worth celebrating. They shared ...
Oligonucleotide Therapeutics – Restoring Sight to the Blind
Vision is one of our most priceless senses. Without it, we miss out on precious ...
Can Oligonucleotides Target RNA Splicing to Treat Disease?
The human body is phenomenally complex, and we are continually discovering incredible facts about it. ...
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 ...
