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, ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
