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, rather than viewing CRISPR as genetic scissors, it may be ...
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 systemic trial for genome editing and the delivery of 2 ...
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 – 100 Years of History Many of you probably enjoyed hearing ...
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 their lives. Sadly, those who receive a diagnosis are the ...
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, Takeda Pharmaceutical, and Ionis Pharmaceuticals may have found a solution. ...
Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics
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 work in some capacity. In a previous article, we delved ...
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 ...
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) ...
2021 Annual Meeting Highlights
We enjoyed another excellent Annual Meeting this year! A more extensive meeting review from 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 ...
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 ...
Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics
Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many ...