Promoters and regulatory elements that improve adeno-associated virus transgene expression in the brain.
Since the first demonstration of central nervous system (CNS) transduction with recombinant adeno-associated virus, improvements in vector production and promoter strength have lead to dramatic increases in the number of cells transduced and the level of expression within each cell. The improvements in promoter strength have resulted from a move away from the original cytomegalovirus (CMV) promoter toward the use of hybrid CMV-based promoters and constitutive cellular promoters. This review summarizes and compares different promoters and regulatory elements that have been used with rAAV as a reference toward achieving a high level of rAAV-mediated transgene expression in the CNS.
Fitzsimons
Helen L
HL
CNS Gene Therapy Center, Department of Neurosurgery, Thomas Jefferson University, Philadelphia, PA 19107, USA.
Bland
Ross J
RJ
During
Matthew J
MJ
D000256:Adenoviridae / Q000235:genetics*; D001921:Brain / Q000821:virology*; D015967:Gene Expression Regulation, Viral; D005809:Genes, Regulator / Q000235:genetics; D011401:Promoter Regions, Genetic / Q000235:genetics*; D019076:Transgenes / Q000235:genetics*