The successful prophylactic treatment of hemophilia A by frequent infusions of plasma concentrates or recombinant factor VIII (hFVIII) indicates that gene therapy may be a potential alternative for the treatment of the disease. For efficient delivery and long-term expression of the hFVIII gene, a novel minimal adenovirus (mini-Ad) vector, MiniAdFVIII, has been developed. The vector is devoid of all viral genes and carries the full-length hFVIII cDNA under the control of the human 12.5-kb albumin promoter. The MiniAdFVIII vector was propagated with the assistance of an ancillary vector in 293 cells and was purified by CsCl banding. Sustained expression of hFVIII at physiologic levels (100-800 ng/mL) was achieved in mice after a single intravenous injection of MiniAdFVIII. The expressed hFVIII had a structure identical to that of recombinant hFVIII, as determined by Western blot analysis. The functionality of the protein was confirmed by the restoration of blood coagulation capacity in MiniAdFVIII-treated hemophilic mice, as determined by tail clipping observations. Although antivector or antihuman FVIII antibodies at various levels were detected, long-term expression of the transgene was observed in the mice that did not generate antibodies against the transgene product. The vector DNA persisted in the liver tissues of the mice with long-term expression. No significant histopathologic findings or toxicities were observed to be associated with the vector in the MiniAdFVIII-treated C57BL/6 mice. These results support the further development of MiniAdFVIII for clinical trials toward the treatment of hemophilia A.

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Kidney transplantation (KT), the optimal treatment for stage 5 chronic kidney disease (CKD), restores impaired fertility in most women of reproductive age. However, infertility occurs in some patients after successful KT. We present our own experience of overcoming secondary tubal infertility by in vitro fertilization (IVF). The patient was a 36-year-old with a transplanted kidney, who had lost two pregnancies in the past due to severe preeclampsia (PE). After the second attempt on cryo-thawed embryo transfer against the background of hormone replacement therapy, one embryo was transferred into the uterus, resulting in pregnancy. Gestational diabetes mellitus (GDM) was diagnosed in the first trimester, and a diet was prescribed. Immunosuppression with tacrolimus, azathioprine and methylprednisolone, prophylaxis of PE with low molecular weight heparin and antiplatelet drugs were administered during pregnancy. Elective cesarean section was performed at 37–38 weeks and a healthy boy was born, weighing 2760 g (25th percentile), 48 cm tall (36th percentile). A stay in the neonatal intensive care unit was not required. The baby is growing and developing normally, the mother’s renal graft function is satisfactory. So, IVF can be successfully used in post-KT patients with infertility issues, provided that the IVF program is carefully controlled, and the pregnancy is managed in a multidisciplinary manner.

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