CRISPR/CAS ALL-IN-TWO VECTOR SYSTEMS FOR TREATMENT OF DMD

The present disclosure provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD), e.g., through ex vivo and in vivo methods of genome editing. The present disclosure also relates to methods and compositions for use of self-inactivating/self-targeting CRISPR/Cas or...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Hauptverfasser:	YANG, Yanfei, POLICE, Seshidhar, Reddy, NG, Robert
Format:	Patent
Sprache:	eng ; fre ; ger
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 SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
Online-Zugang:	Volltext bestellen
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Beschreibung
Zusammenfassung:	The present disclosure provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD), e.g., through ex vivo and in vivo methods of genome editing. The present disclosure also relates to methods and compositions for use of self-inactivating/self-targeting CRISPR/Cas or CRISPR/Cpf1 systems to genetically modify cells, e.g., to modulate the expression, function, and/or activity of the dystrophin gene.