MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES

Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing. L'invention concerne des substances et des méthode...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Hauptverfasser:	LIN, Michelle, I-ching, MISHRA, Bibhu, Prasad, COWAN, Chad, Albert, LUNDBERG, Ante, Sven, KERNYTSKY, Andrew, BORLAND, Todd, Douglass, CHAKRABORTY, Tirtha, PAIK, Elizabeth, Jae-eun
Format:	Patent
Sprache:	eng ; fre
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 PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
Online-Zugang:	Volltext bestellen
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Beschreibung
Zusammenfassung:	Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing. L'invention concerne des substances et des méthodes pour traiter un patient atteint d'une hémoglobinopathie, à la fois ex vivo et in vivo, ainsi que des substances et des méthodes pour effacer, moduler ou inactiver une séquence de contrôle transcriptionnel dans un gène BCL1 1 A dans une cellule par édition génomique.