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Corrección de un defecto genético mediante trasplante nuclear y terapia celular y génica combinada

William M Rideout1, Konrad Hochedlinger, Michael Kyba

  • 1Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA.

Cell
|April 17, 2002
PubMed
Resumen

Este estudio demuestra un nuevo enfoque de terapia génica para la inmunodeficiencia mediante la reparación del gen Rag2 en las células madre embrionarias. Este método restauró con éxito la función inmune en ratones, ofreciendo un tratamiento potencial para trastornos genéticos.

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