作者: Brendan Murphy
DOI: 10.1007/978-94-017-0926-2_19
关键词: Somatic cell 、 Clinical trial 、 Gene delivery 、 Viral vector 、 Animal origin 、 Biology 、 Gene 、 Infectious disease (medical specialty) 、 Genetic enhancement 、 Computational biology
摘要: Gene therapy has recently received attention as a novel strategy for the correction of human disease at genetic level. Several methods transferring genes into somatic mammalian cells have been developed and led to range gene clinical trials both inherited acquired disorders. The majority used viral vectors animal origin mediate transfer, with retroviruses, adenoviruses adeno-associated viruses constituting vehicles choice. While principle proven, appropriate therapeutic responses being noted, its potential not yet realised in large scale trials. Critical areas future development remain design more efficient, safer, delivery. Viral are currently undergoing extensive modifications produce custom-made particular applications.