We demonstrated that transduction of SCD BM CD34+ cells with this vector reduced deoxygenation-induced sickling of in vitro-derived RBC progeny.31 To further support use of the V5m3-400 vector in SCD clinical trials, we tested whether SCD mice receiving nonablative irradiation would engraft with γ-globin gene-corrected HSCs to a level sufficient for a therapeutic effect. This evidence concerns the gene CD34 and Schnyder corneal dystrophy.