Newly Approved Drug Shows Clinical and Quality-of-Life Improvements for HAE Patients
Emily Wheeler was 19 when she noticed a gradual swelling in her left foot. Believing it was an allergic reaction, she went to her doctor, who told her it was gout and gave her several pills. An hour later, she started vomiting uncontrollably and had excruciating abdominal pain. She went to the hospital, where they stopped her vomiting, and ...
OTS Member Highlight – Dr. Richard Geary
Dr. Richard Geary, current OTS President, has always been passionate about health and finding ways to improve people's well-being. Born with a rare disease, he was in the medical spotlight at a young age; now, he’s known for his incredible contributions to the scientific field and his enduring work as the executive vice president and chief development officer at Ionis ...
Binding and Ligand Activation Driven Enrichment–Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency
Authors: Telmo Llanga, Korie Bush, Ying Sun, Amy Yan, Jonathan Zhou, Jan Gorodkin, and Bruce A. Sullenger AbstractClustered regularly interspaced short palindromic repeats-based editing is inefficient at over two-thirds of genetic targets. A primary cause is ribonucleic acid (RNA) misfolding that can occur between the spacer and scaffold regions of the gRNA, which hinders the formation of functional Cas9 ribonucleoprotein ...
Targeting ALS-FUS: Early Insights from the Jacifusen (ION363) Case Series
Amyotrophic lateral sclerosis (ALS) remains one of the most devastating diseases and is the most common motor disease among adults, affecting around 55,000 people in the US, Europe, and Japan. More than 40 genes have been linked to ALS, providing a valuable opportunity for targeted treatment. Among these, mutations in the FUS gene cause a particularly aggressive form of ...
Behind the Rapid Development of an Individualized CRISPR Therapy for a Deadly Rare Disease
A baby is the first person to receive a bespoke CRISPR therapy designed specifically for him that directly fixes a disease-causing mutation in his genome. It wasn't long after KJ was born in the summer of 2024 that doctors noticed something was wrong. Within 48 hours, he was lethargic and struggled to breathe, prompting doctors to run a blood ...
The Oligonucleotide Therapeutics Society Announces the 21st Annual Meeting in Budapest, Hungary
The Oligonucleotide Therapeutics Society (OTS) is pleased to announce the 21st Annual OTS Meeting, scheduled for October 19–22, 2025, at the Budapest Congress Center in Budapest, Hungary. This premier global gathering of the oligonucleotide community is renowned for fostering dynamic, cross-disciplinary dialogue—bringing together leaders in chemistry, delivery, biology, immunology, and clinical medicine to advance the field of oligonucleotide therapeutics. ...
Newly Approved Drug Shows Clinical and Quality-of-Life Improvements for HAE Patients
Emily Wheeler was 19 when she noticed a gradual swelling in her left foot. Believing ...
OTS Member Highlight – Dr. Richard Geary
Dr. Richard Geary, current OTS President, has always been passionate about health and finding ways to ...
Binding and Ligand Activation Driven Enrichment–Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency
Authors: Telmo Llanga, Korie Bush, Ying Sun, Amy Yan, Jonathan Zhou, Jan Gorodkin, and Bruce A. ...
Targeting ALS-FUS: Early Insights from the Jacifusen (ION363) Case Series
Amyotrophic lateral sclerosis (ALS) remains one of the most devastating diseases and is the most ...
Behind the Rapid Development of an Individualized CRISPR Therapy for a Deadly Rare Disease
A baby is the first person to receive a bespoke CRISPR therapy designed specifically for ...
The Oligonucleotide Therapeutics Society Announces the 21st Annual Meeting in Budapest, Hungary
The Oligonucleotide Therapeutics Society (OTS) is pleased to announce the 21st Annual OTS Meeting, scheduled ...
