Haema 2016; 7(1): 57-71
by George Vassilopoulos1,2, Manolis Simantirakis1
1Affiliate Investigator Cell and Gene Therapy Laboratory, BRFAA, Athens,
2Division of Hematology, Larisa University Hospital, University of Thessaly, Greece
Abstract
Gene transfer technology has been developed based on retroviruses, AAV and adeno-viruses. The basic principle for every vector development is based on the delineation of the regulatory in cis sequences and the helper in trans functions. Next technological steps have focused on the choice of proper promoters and chromatin insulators. Although vector improvements over the last 30 years are substantial and have led to clinical trials, the technology is still costly and has significant side effects from insertional mutagenesis. This has resulted in a shift towards gene editing technology and the use of vectors that can mediate genetic correction in situ (such as AAV).