Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...

Researchers have unveiled a new DNA-based therapy that silences the PCSK9 gene to lower cholesterol without statin side effects. Early results show dramatic reductions in cholesterol levels, offering ...

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

A significant advance in treatment of patients with immunity to AAVs previously ineligible for clinical trials and existing ...

Significant advancements in gene silencing and gene editing offer new hope for treating rare disorders. Techniques like RNA ...

The gene therapy for neurological diseases market is poised for growth due to rising neurological disorder prevalence, demand ...

Endocrine therapy typically slows cell cycle progression by reducing cyclin D1, which partners with CDK4/6 to promote cell ...

Mercer County Community College launches an aseptic biomanufacturing program and mobile lab in Trenton to train students for ...

Researchers have understood for decades that some forms of deafness are inherited, yet until now, few of those genetic clues ...

Engineers have created DIAL, a modular system that precisely sets and edits protein levels in synthetic gene circuits. By ...

A young Massapequa girl was diagnosed with PKAN, a progressive disease that will affect her ability to walk and talk as she ...