Lentiviral vectors have emerged as indispensable tools in gene therapy, offering the ability to integrate therapeutic genes into both dividing and non-dividing cells. Their unique capacity to achieve ...

The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...

The FDA suspends Regenxbio gene therapy trials for Hurler and Hunter syndromes over safety concerns after a participant developed a brain tumor.

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, ...

(RTTNews) - Sarepta Therapeutics (SRPT), Wednesday announced that the FDA has designated its rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 for limb-girdle muscular dystrophy ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...