Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

Ryuji Morizane, MD, Ph.D., of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read more at straitstimes.com. Read more at straitstimes.com.

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

After a patient died following a high dose of Neurogene’s Rett syndrome gene therapy candidate, the biotech is sharing details about the event and the new safety protocols being incorporated into the ...

Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults with hemophilia B who currently use factor IX prophylaxis therapy for blood ...

Asimov launched its AAV Edge Stable Producer system with the goal of addressing the shortcomings of traditional transient transfection-based production and bringing AAV manufacturing standards closer ...

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV ...