Natural gene therapy in monozygotic twins with Fanconi anemia

Monozygotic twin sisters, with nonhematologic symptoms of Fanconi anemia (FA), were discovered to be somatic mosaics for mutations in the FANCA gene. Skin fibroblasts, but not lymphocytes or committed hematopoietic progenitors, were sensitive to DNA cross-linking agents. Molecular analysis revealed,...

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Bibliographische Detailangaben
Hauptverfasser: Mankad, Anuj, Taniguchi, Toshiyasu, Cox, Barbara, Akkari, Yassmine, Rathbun, R. Keaney, Lucas, Lora, Bagby, Grover, Olson, Susan, D'Andrea, Alan, Grompe, Markus
Format: Artigo
Sprache:Inglês
Veröffentlicht: The American Society of Hematology 2006
Schlagworte:
Gene Therapy
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1895746/
https://ncbi.nlm.nih.gov/pubmed/16397136
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2005-07-2638
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