Non-replicating recombinant vector derived from adeno-associated virus AAV6 serotype, containing a codon-optimised version of the human Factor VIII gene
Non-replicating recombinant vector derived from adeno-associated virus AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene
NKG2D-chimeric antigen receptor, the truncated CD19 tag and the TIM8 molecule which interferes with the interaction between the natural TCR and endogenous CD3ζ