Recent Articles2022-03-14T07:39:44+00:00
25August, 2021

Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

Categories: Featured Perspectives On Current Science|

Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these problems and there are multiple solutions that have proven to [...]

9August, 2021

OTS President’s Paper Pick – August 2021

Categories: Presidents Pick, Perspectives on Current Science|

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, [...]

6August, 2021

Lipid Nanoparticles: Nanomedicine’s Triumph

Categories: Perspectives on Current Science|

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 [...]

1August, 2021

A Momentous CRISPR Milestone was Achieved with Remarkable Results

Categories: Perspectives on Current Science, Featured Perspectives On Current Science|

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, [...]

28June, 2021

Oligonucleotide Therapeutics – Restoring Sight to the Blind

Categories: Perspectives on Current Science|

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 [...]

21June, 2021

Can Oligonucleotides Target RNA Splicing to Treat Disease?

Categories: Perspectives on Current Science|

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 [...]

29May, 2021

mRNA Is Capable of Providing Solutions Far Beyond Vaccines

Categories: Perspectives on Current Science|

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 [...]

20May, 2021

Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

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 walk. Ultimately, the disease would [...]

14April, 2021

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

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 on emergent safety concerns, the [...]

Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

August 25th, 2021|Categories: Featured Perspectives On Current Science|

Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for [...]

Comments Off on Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

A Momentous CRISPR Milestone was Achieved with Remarkable Results

August 1st, 2021|Categories: Perspectives on Current Science, Featured Perspectives On Current Science|

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 [...]

Comments Off on A Momentous CRISPR Milestone was Achieved with Remarkable Results

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 [...]

Comments Off on Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

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 [...]

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

Get the Latest News!

  • Category

Go to Top