Efficient breeding system of infertile Niemann-Pick disease type C model mice by in vitro fertilization and embryo transfer.

Abstract

Niemann-Pick disease type C (NPC) is a lethal genetic disease with mutations inorgene.-deficient () mice have been used as a model for NPC pathogenesis to develop novel therapies for NPC. However,mice are infertile; thus, securing sufficient numbers for translational research is difficult. Hence, we attempted reproductive engineering techniques such as in vitro fertilization (IVF) and sperm cryopreservation. For the first time, we succeeded in producing fertilized oocytes via IVF using male and femalemice. Fertilized oocytes were also obtained via IVF using cryopreserved sperm frommice. The obtained fertilized oocytes normally developed into live pups via embryo transfer, and they eventually exhibited NPC pathogenesis. These findings are useful for generating an efficient breeding system that overcomes the reproductive challenges ofmice and will contribute to developing novel therapeutic methods using NPC model mice.