Small Nuclear RNAs (snRNAs) Enhance RNA Base Editing
Gene editing technologies are transforming molecular medicine, offering unprecedented potential to treat — and even cure — a range of genetic disorders. However, as tools like CRISPR enter clinical settings, concerns about its safety, specificity, and long-term side effects, such as off-target edits or increased cancer risk, have prompted scientists to improve the technology or find alternatives. With these ...
Leveraging Nature’s Carrier: Albumin-Binding Dendritic siRNA for Enhanced Tumor Targeting
Small interfering RNA (siRNA) therapeutics are rapidly reshaping the landscape of precision medicine, offering the ability to silence disease-driving genes with exceptional specificity and durability. However, due to delivery barriers, the promise of siRNA therapy has largely been confined to the liver. Recent advances and mounting efforts are being poured into expanding siRNAs extrahepatically, to enable therapies for diseases ...
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 ...
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 ...
Small Nuclear RNAs (snRNAs) Enhance RNA Base Editing
Gene editing technologies are transforming molecular medicine, offering unprecedented potential to treat — and even ...
Leveraging Nature’s Carrier: Albumin-Binding Dendritic siRNA for Enhanced Tumor Targeting
Small interfering RNA (siRNA) therapeutics are rapidly reshaping the landscape of precision medicine, offering the ...
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 ...
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 ...
