Perspectives on Current Science

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, she received her first kidney transplant. Gutierrez was born with polycystic kidney disease (PKD), a genetic condition that makes fluid-filled cysts grow on the kidney and can lead to ...

In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). The approvals — the second largest in 30 years — include an oligonucleotide therapy for blood cancer, an antisense oligonucleotide shown to ...

Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not ...

At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn't stop crying and wasn't eating. Concerningly, her triglyceride levels were in the 20,000's, far over the normal 150 level. Darlene ...