Stanford researchers have developed a genome engineering platform for generating genetically precise human cellular models of Lowe syndrome, a rare inherited disorder frequently associated with severe ocular complications, including early-onset glaucoma.
Stanford scientists have developed a gene integration system that uses human-derived helicases paired with CRISPR technology to enable precise insertion of long DNA sequences at targeted genomic locations.
Stanford researchers have developed a new gene editing approach that enables red blood cell-specific gene expression for the treatment of enzyme deficiencies.
Stanford researchers have engineered retroviral and virus-like delivery systems for producing universal pseudotyped vehicles for cell and gene therapies.
Introduction: Blood cell transfusion plays a vital role in modern medicine–supporting surgery, obstetrics, trauma care, and cancer chemotherapy. In the US alone, more than 12 million red-cell units are consumed annually.
Stanford inventors have engineered an adeno-associated virus (AAV) variant on the existing LK03 platform that enables this highly efficient primate-specific serotype for use in rodent preclinical studies.