Sentences with phrase «previous gene therapy trials»

The vector is a self - inactivating lentivirus — a member of a family of viruses that can insert genes into mammalian cells and drive expression of those genes — that has been engineered to avoid triggering the development of leukemia, a complication seen in previous gene therapy trials for immunodeficiency syndromes, including WAS.

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

Not exact matches

Previous hemophilia gene therapy trials have been frustrated by an immune response to the gene therapy product that limited the success of the therapy.

The researchers used the new technique to mutate the genes CXCR4 and CCR5, which encode receptor molecules that different strains of the HIV virus use to sneak in and infect immune cells and which have been targeted in previous cell therapy trials.

A new animal study involving virally - delivered gene therapies has indicated that high treatment doses might not be as safe as previous human trials have suggested