Article

Complete correction of murine phenylketonuria by selection-enhanced hepatocyte transplantation

2023-08-28

Abstract excerpt

Hepatocyte transplantation for genetic liver diseases has several potential advantages over gene therapy. However, low efficiency of cell engraftment has limited its clinical implementation. This problem could be overcome by selectively expanding transplanted donor cells until they replace enough of the liver mass to achieve therapeutic benefit. We previously described a gene therapy method to selectively expand h...

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Literature Corpus work: 48523f99-f3d1-57b0-b9df-6aaf3964fe9f
DOI: 10.1101/2023.08.27.554228

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