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 ...
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, ...
Genome Sequencing and Gene Editing: Ancient DNA in a Living Species
Known for their large size — sometimes growing up to six feet in length — dark fur and bone-cracking back teeth, dire wolves used to be the most prominent species in continental North America (1). The canines became extinct roughly 10,000 years ago, gradually dying out as their prey ...
OTS Member Highlight – Masad Damha, PhD
For Masad Damha, falling in love with science was easy. Growing up in Managua, Nicaragua, his high school teachers encouraged scientific exploration, curiosity, and experimentation, such as processing vegetable oil into soap, producing hydrogen and oxygen from water via electrolysis, and determining the gravitational force constant using a pendulum. ...
Targeting DNA Mismatch Repair as a Potential Therapeutic Strategy for Huntington’s Disease
Huntington's disease (HD) is a devastating and fatal neurodegenerative disorder affecting movement, thinking, and behavior. In the United States alone, there are approximately 40,000 people with symptomatic Huntington's disease, and more than 200,000 are at risk of inheriting it. The fatal disease often leaves a devastating family legacy, as ...
FDA Approval of Vutrisiran Brings New Treatment for ATTR-CM
When Sean Riley started getting numbness in his hands, he saw a local surgeon who performed carpal tunnel surgery on both his wrists. At the time, he didn't think much of his symptoms, knowing carpal tunnel was common, he attributed his symptoms to the typing he occasionally did. Then, ...
OTS Member Highlight – David Corey, PhD
Former Oligonucleotide Therapeutics Society (OTS) President David Corey grew up being inspired by his father's love of science, noting that his father was not only good at what he did but he also enjoyed it. "I never measured myself against him because I knew I could not expect to ...
NanoCas: A Newly Identified Compact Nuclease May Expand CRISPR’s Reach
The revolutionary CRISPR-Cas system has proven to be a precise method for genome editing, showing great promise in tackling genetic disorders. However, clinical trials for CRISPR-based treatments have faced delivery challenges due to the large size of the editing system. In the quest to overcome these limitations, researchers recently ...
OTS Member Highlight – Holly Kordasiewicz, PhD
Holly Kordasiewicz was a curious, chatty child with a penchant for asking questions. At dinner, conversations were often about the fascinating things her mom, a registered nurse, learned at work, sparking Kordasiewicz's interest in medicine and biology. As her grandparents aged, and she witnessed neurodegenerative diseases gradually and relentlessly ...
OTS Member Highlight – Dr. Annemieke Aartsma-Rus
Growing up the oldest of four children in Leiden, Netherlands, Dr. Annemieke Aartsma-Rus became fascinated with medical research and genetics as a teenager. While her father's work as a physician made her realize she didn't want to be a doctor, it did teach her that there were numerous diseases ...
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 ...
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 ...
Genome Sequencing and Gene Editing: Ancient DNA in a Living Species
Known for their large size — sometimes growing up to six feet in length — dark fur and bone-cracking back teeth, dire wolves used to be the most prominent ...
OTS Member Highlight – Masad Damha, PhD
For Masad Damha, falling in love with science was easy. Growing up in Managua, Nicaragua, his high school teachers encouraged scientific exploration, curiosity, and experimentation, such as processing vegetable ...
Targeting DNA Mismatch Repair as a Potential Therapeutic Strategy for Huntington’s Disease
Huntington's disease (HD) is a devastating and fatal neurodegenerative disorder affecting movement, thinking, and behavior. In the United States alone, there are approximately 40,000 people with symptomatic Huntington's disease, ...
FDA Approval of Vutrisiran Brings New Treatment for ATTR-CM
When Sean Riley started getting numbness in his hands, he saw a local surgeon who performed carpal tunnel surgery on both his wrists. At the time, he didn't think ...
OTS Member Highlight – David Corey, PhD
Former Oligonucleotide Therapeutics Society (OTS) President David Corey grew up being inspired by his father's love of science, noting that his father was not only good at what he ...
NanoCas: A Newly Identified Compact Nuclease May Expand CRISPR’s Reach
The revolutionary CRISPR-Cas system has proven to be a precise method for genome editing, showing great promise in tackling genetic disorders. However, clinical trials for CRISPR-based treatments have faced ...
OTS Member Highlight – Holly Kordasiewicz, PhD
Holly Kordasiewicz was a curious, chatty child with a penchant for asking questions. At dinner, conversations were often about the fascinating things her mom, a registered nurse, learned at ...
OTS Member Highlight – Dr. Annemieke Aartsma-Rus
Growing up the oldest of four children in Leiden, Netherlands, Dr. Annemieke Aartsma-Rus became fascinated with medical research and genetics as a teenager. While her father's work as a ...