Viral vectors comprising RDH12 coding regions and methods of treating retinal dystrophy

The present invention relates to viral vectors comprising RDH12 coding regions and methods of treating retinal dystrophy. Materials, methods, and uses are provided for treating ophthalmic conditions, such as Leber's congenital amaurosis, by administering an effective amount of an adeno-associat...

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Bibliographische Detailangaben
Hauptverfasser:	THOMPSON DAVID ALLEN, SMITH ANDREW JOHN, ALI ROBIN R
Format:	Patent
Sprache:	chi ; 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 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 relates to viral vectors comprising RDH12 coding regions and methods of treating retinal dystrophy. Materials, methods, and uses are provided for treating ophthalmic conditions, such as Leber's congenital amaurosis, by administering an effective amount of an adeno-associated virus AAV2, serotype 5 (AAV 2/5), or AAV-5 comprising an expressible coding region of human RDH12. 本发明涉及包括RDH12编码区的病毒载体和治疗视网膜营养不良的方法。提供了用于通过施用有效量的包括人RDH12的可表达编码区的腺相关病毒AAV2、血清型5(AAV 2/5)或AAV-5治疗如莱伯氏先天性黑蒙等眼科病状的材料、方法和用途。