Schambach, A.

vector expression therapy lentiviral level b cells number spleen figure transplantation blood gene therapy promoter transduced b-cell reconstitution ficiency il 2rg mice leukemia integration t-cell analysis sffv.corag γ cpr-co γc serum patient btk à/à mice lentiviral vectors group antibody rag 1 expression wild-type protein 1-/- t cells animal spleen cells ficiency yy ng transfer response development transgene scid-x ficient btk expression recipient cpr-co immunode fl ow cytometry control sffv.corag 1-treated mice netherland 19.cobtk lentiviral gene therapy sequence efs.cobtk correction mouse pretransplant conditioning lentiviral gene transfer result rag 1à/à mice cd 19.cobtk transduction conditioning codon-optimized virus b-cell development btk à/à rag 1 immunode ficiency ucoe.corag codon kinase american society population tyrosine 1-treated insertional mutagenesis wild-type cells