Gene Therapy
Researchers uncover RNA hubs driving kidney cancer growth
Scientists at Texas A&M University Health Science Center have discovered that RNA forms droplet-like hubs in translocation renal cell carcinoma, activating tumor genes. By developing a molecular switch to dissolve these hubs, they halted cancer growth in lab and mouse models. The findings, published in Nature Communications, offer a new approach for treating this aggressive pediatric cancer.
New DNA-based therapy reduces cholesterol by nearly 50 percent
Researchers from the University of Barcelona and the University of Oregon have developed a DNA-based treatment that targets the PCSK9 gene to lower cholesterol levels without the side effects of statins. Using polypurine hairpins, the therapy increases cholesterol uptake by cells and reduces artery-clogging lipids in animal models. The findings, published in Biochemical Pharmacology, suggest a safer alternative for preventing cardiovascular diseases.
Nanomachines Target Senescent Cells in Reproduction
Targeted nanomachines for clearing senescent cells in reproductive tissues, along with gene therapies for telomerase reactivation, were reported on September 11, 2025.
FDA Grants Accelerated Approval for First Barth Syndrome Treatment
The U.S. Food and Drug Administration has granted accelerated approval to Forzinity, the first treatment for Barth syndrome, a rare X-linked genetic disorder primarily affecting males. Developed by Stealth Biotherapeutics, the therapy targets mitochondrial dysfunction underlying the condition, offering new hope for patients with this life-threatening disease. This milestone follows years of advocacy and highlights progress in addressing ultra-rare disorders.