Mei’s Story: Investigation Reveals Unreported Fatal Outcome After In Vivo Base Editing of CHD3
In March 2025, a six-year-old girl received the first reported in vivo base-editing treatment directed at the human CNS. The girl, given the pseudonym “Mei” — meaning “beautiful” in Chinese — had Snijders Blok-Campeau syndrome, an extremely rare genetic condition associated with mutations in the CHD3 gene. Her condition was not life-threatening, and she was on the milder side ...
Roche Ends Two HD Clinical Trials as Other Huntingtin-Lowering Therapies Advance
On July 9, Roche announced it would discontinue two ongoing clinical studies investigating treatments for Huntington's disease (HD) after separate analyses showed the programs would not achieve their intended goals. While the news is a significant disappointment for the HD community, both studies have provided valuable insights that are expected to shape future research. Understanding Huntington's Disease Huntington's disease ...
Antisense Oligonucleotide Therapy in Chronic Hepatitis B: Clinical Progress and Phase 3 Outcomes
When Sheree was 26, she kept wondering why she was always so tired. Though she was a mom and a nurse working multiple double shifts, she was still young and knew she shouldn’t be this fatigued. Then the nausea and abdominal pain began. Soon, the only things she could tolerate were burnt toast, oatmeal, and water. When she woke up ...
Al3Cas12f RKK: A New, Enhanced Compact Editor that is Highly Efficient
CRISPR-based gene-editing tools have transformed the biomedical field, but delivering these systems directly into human tissues — known as in vivo gene editing — remains a major challenge. Many therapeutic CRISPR approaches still rely on ex vivo editing, where the patient’s cells are removed, edited outside the body, and reinfused. This method is used partly because commonly used CRISPR ...
OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he wanted to be a scientist when he grew up. Now, decades later, Dr. Keith Gagnon is a biochemist and molecular biologist specializing in RNA biology, nucleic acid therapeutics, repeat expansion disorders, and CRISPR-Cas systems, serving as an Associate Professor in the Department of Biochemistry ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because of their ability to target virtually any disease-related gene product, several ASO drugs have been approved, and many more are in development (1). While they remain among the most precise therapies, investigations are ongoing to find improvements and increase safety. A recent study from ...
Mei’s Story: Investigation Reveals Unreported Fatal Outcome After In Vivo Base Editing of CHD3
In March 2025, a six-year-old girl received the first reported in vivo base-editing treatment directed ...
Roche Ends Two HD Clinical Trials as Other Huntingtin-Lowering Therapies Advance
On July 9, Roche announced it would discontinue two ongoing clinical studies investigating treatments for ...
Antisense Oligonucleotide Therapy in Chronic Hepatitis B: Clinical Progress and Phase 3 Outcomes
When Sheree was 26, she kept wondering why she was always so tired. Though she was ...
Al3Cas12f RKK: A New, Enhanced Compact Editor that is Highly Efficient
CRISPR-based gene-editing tools have transformed the biomedical field, but delivering these systems directly into human ...
OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because ...
