Zilganersen Trial Shows Slowed Disease Progression and Improvement in Gait Speed
Hailey was born a healthy baby, her mom says, hitting all her infant milestones like rolling over and sitting up. But at nine months old, she began falling over when she sat up and having constipation and little sleep. Her concerned mom brought it up at a pediatric appointment, but she was dismissed as being a nervous first-time parent. ...
Targeting APOC3: Redemplo Joins the Growing Landscape of Treatment for FCS
When Julie was just 18, her blood results revealed alarmingly high triglyceride levels at over 1,000 mg/dL. Two years later, she had her first acute pancreatitis attack. Although she would suffer from monthly pancreatitis flares after having her son and her triglycerides would escalate to over 10,000 mg/dL, she wouldn’t receive a diagnosis of familial chylomicronemia syndrome (FCS) for ...
TfR1-Targeted Bicyclic Peptide-Oligonucleotide Conjugates for Improved Potency and Enhanced Muscle Delivery
Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are uniquely suited to address single-gene diseases affecting skeletal and cardiac muscles. While distributed broadly, they are more likely to accumulate in the liver and kidneys than muscles, thus limiting their treatment applicability. Additionally, the anticipated dose levels needed for unconjugated ASOs to reach these muscles are very high. Ionis Pharmaceuticals, ...
OTS Member Highlight – Dr. Rebecca Miles
The incoming OTS President, Dr. Rebecca Miles, has always been curious about the way things work, and in high school, a human anatomy and physiology class sparked her passion for researching human diseases. As the first person in her family to pursue higher education, she was initially unsure which career would be the best path for understanding the human ...
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 ...
Zilganersen Trial Shows Slowed Disease Progression and Improvement in Gait Speed
Hailey was born a healthy baby, her mom says, hitting all her infant milestones like ...
Targeting APOC3: Redemplo Joins the Growing Landscape of Treatment for FCS
When Julie was just 18, her blood results revealed alarmingly high triglyceride levels at over ...
TfR1-Targeted Bicyclic Peptide-Oligonucleotide Conjugates for Improved Potency and Enhanced Muscle Delivery
Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are uniquely suited to address single-gene diseases ...
OTS Member Highlight – Dr. Rebecca Miles
The incoming OTS President, Dr. Rebecca Miles, has always been curious about the way things ...
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 ...
