Article
Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy.
Cell - 5 Apr 2002
Rideout William M, Hochedlinger Konrad, Kyba Michael, Daley George Q, Jaenisch Rudolf
Abstract excerpt
Immune-deficient Rag2(-/-) mice were used as nuclear donors for transfer into enucleated oocytes, and the resulting blastocysts were cultured to isolate an isogenic embryonic stem cell line. One of the mutated alleles in the Rag2(-/-) ES cells was repaired by homologous recombination, thereby restoring normal Rag2 gene structure. Mutant mice were treated with the repaired ES cells in two ways. (1)...
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