COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

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Bibliographische Detailangaben
Hauptverfasser: BOITANO, Anthony Edward, STEVENSON, Susan C, SNEAD, Jennifer, YANG, Yi, COOKE, Michael, MULUMBA, Kabungo, KLICKSTEIN, Lloyd B, POLICE, Seshidhar Reddy, LESCARBEAU, Reynald, MICKANIN, Craig Stephen, STEWART, Morag
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.