Does CRISPR Gene Editing Increase the Risk of Developing Cancer?
CRISPR is a useful tool in a wide array of applications, and one of the most headline-grabbing is its potential in gene editing. As with any new technology or medication, there are inherent risks that must be discovered and mitigated. This is especially true in the arena of gene-editing, as ...
Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease
It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a treatment that produced significant results ...
Looking back… Looking forward
This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) will take over as President and Richard Geary (Ionis Pharmaceuticals) will become the Society’s next President-Elect. As we delayed this ...
Can an ASO Reduce Disease Progression in ALS?
What if you noticed your body acting a bit “off,” doing something unusual and more than a bit worrying? Then, you go to a doctor and are eventually told that you have a debilitating neurodegenerative disease and can expect to progressively lose control of your muscles. Your quality of life ...
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics – a platform that could allow for administration of siRNA medications just once a year with highly potent knockdown of ...
New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells
CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could result in a more versatile tool that could greatly improve and expand CRISPR-based therapeutics. As Dr. Stanley Qi pointed out, ...
President’s Pick of Presentations from the Annual OTS Meeting
By: Annemieke Aartsma-Rus, Ph.D. The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges the pandemic poses on research and development in general, the oligonucleotide therapy field has made many advances, including the first ...
2021 Annual Meeting Highlights
We enjoyed another excellent Annual Meeting this year! A more extensive meeting review from the organizing committee will be forthcoming, so be sure to watch for it. In the meantime, here is a broad overview highlighting a few of the many fascinating and intriguing presentations. Bob Letsinger, PhD – ...
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 to minimize the impact on ...
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 Tokyo Medical and Dental University, ...
Does CRISPR Gene Editing Increase the Risk of Developing Cancer?
CRISPR is a useful tool in a wide array of applications, and one of the most headline-grabbing is its potential in gene editing. As with any new technology or medication, ...
Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease
It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs ...
Looking back… Looking forward
This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) ...
Can an ASO Reduce Disease Progression in ALS?
What if you noticed your body acting a bit “off,” doing something unusual and more than a bit worrying? Then, you go to a doctor and are eventually told that ...
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics ...
New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells
CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could ...
President’s Pick of Presentations from the Annual OTS Meeting
By: Annemieke Aartsma-Rus, Ph.D. The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges ...
2021 Annual Meeting Highlights
We enjoyed another excellent Annual Meeting this year! A more extensive meeting review from the organizing committee will be forthcoming, so be sure to watch for it. In the ...
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 ...
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 ...
