AAV6-iCasp9

AAV6-iCasp9 is a gene therapy construct that utilizes an adeno-associated virus serotype 6 (AAV6) vector to deliver an inducible Caspase 9 (iCasp9) suicide gene. This system is designed as a safety mechanism for cell-based therapies, particularly those involving hematopoietic stem cells (HSCs) or T cells, allowing for the selective elimination of modified cells in the event of severe adverse effects such as graft-versus-host disease (GvHD) or cytokine release syndrome. The iCasp9 transgene encodes a fusion protein consisting of human Caspase 9 and a modified FK506-binding protein (FKBP12) domain. Upon administration of a small-molecule chemical inducer of dimerization (CID), such as rimiducid (AP1903), the iCasp9 proteins undergo dimerization, which triggers the apoptotic cascade and leads to rapid cell death. AAV6 is specifically employed due to its high efficiency in transducing human CD34+ hematopoietic stem and progenitor cells, often serving as a donor template for homology-directed repair (HDR) in genome-editing workflows.

Other names

AAV6-inducible Caspase 9AAV-6-inducible Caspase 9AAV 6-inducible Caspase 9AAV6-iCaspase9AAV-6-iCaspase9AAV 6-iCaspase9