In utero Transplantation: Potential for Humanized Muscle in Animals

Abstract

Multiple therapeutic avenues are being investigated to treat muscular dystrophies and myopathies but many of them are met with limitations that are hard to overcome with existing technology. Moreover, animal models that are commonly used to study these diseases do not sufficiently recapitulate the severity of the disease in humans. We have attempted to develop a new technique that could alleviate these issues. In utero transplantation has been successfully used in the treatment of hematopoietic disorders, but their potential to generate chimeras in other organs has been less frequently investigated. We utilized an ex vivo culture system for whole embryos as a proof of concept to determine whether in utero transplantation would be a suitable method to generate chimerism in muscle. We then transplanted cells in utero into the developing muscle of mouse embryos. We were able to detect the presence of transplanted cells up to three days post-transplantation. ES cell derived progenitors also showed similar results. We therefore propose that in utero transplantation can be used to potentially generate humanized muscle in various animals for therapeutic or scientific purposes.