"Proof of principle", Gene therapy by allele selection in a mouse model of beta-thalassemia

J Clin Invest. 2011;121(2):623–627. doi:10.1172/JCI45377.(February 1, 2011).

Sigrid Eckardt1, N. Adrian Leu2, Ashley Yanchik2, Seigo Hatada3, Michael Kyba4 and K. John McLaughlin1,5

1Center for Molecular and Human Genetics, The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio, USA.
2University of Pennsylvania School of Veterinary Medicine, Philadelphia, Pennsylvania, USA.
3Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, North Carolina, USA.
4Lillehei Heart Institute and Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
5Department of Pediatrics, College of Medicine, The Ohio State University, Columbus, Ohio, USA.

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"genetic correction strategy without gene targeting"

"In the case of recessive diseases, MHC-homozygous, disease-free PG ES cells derived from donated oocytes from a heterozygous mother may also generate useful pluripotent cells for treatment in their affected offspring."