GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA

The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.

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Bibliographische Detailangaben
Hauptverfasser:	Sanz, Jose Carlos Segovia, Roncero, Juan Antonio Bueren, Galdo, Paula Rio, Murillo, Africa Gonzalez, Ordóñez, Susana Navarro, Olea, Jose Antonio Casado, Navarro, Julian Sevilla, Amurrio, Guillermo Güenechea
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.