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Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications

Eur J Pharm Biopharm. 2015; 
Sch?n C, Biel M, Michalakis S.
Products/Services Used Details Operation
PCR Cloning and Subcloning … In-Fusion Cloning Kit from Takara (Clontech #639636) and GenBuilder DNA Assembly from GenScript (#L00701-10 or L00744-10) work well (see Note 3) After verifying by either enzyme digestion or sequencing analysis, DNA is prepared by an endo-free plasmid preparation … Get A Quote

摘要

Adeno-associated virus (AAV) vectors are the most widely used vehicle systems for neuronal gene transfer. This popularity is based on the non-pathogenic nature of AAVs and their versatility making them a multifunctional vector system for basic research and clinical applications. AAVs are successfully applied in clinical and pre-clinical gene therapy studies for inherited retinal disorders. Their excellent transduction profile and efficiency also boosted the use of AAV vectors in basic research. The AAV vector system can be easily modified and adjusted at multiple levels to allow for optimized and specific gene expression in target cells. Here, we will provide an overview on the AAV vector system and its applica... More

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