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Adeno-Associated Virus as Gene Delivery Vehicle into the Retina.

Methods Mol Biol. 2020; 
Deng S1, Oka K2.
Products/Services Used Details Operation
PCR Cloning and Subcloning … In-Fusion Cloning Kit from Takara (Clontech #639636) and GenBuilder DNA Assembly from<br> <b>GenScript</b> (#L00701-10 or L00744-10) work well (see Note 3). After verifying by either enzyme<br> digestion or sequencing analysis, DNA is prepared by an endo-free plasmid preparation … Get A Quote

摘要

Initially discovered as a contaminant of adenovirus preparations, adeno-associated virus (AAV) has proved one of the most promising viral vectors for human gene therapy. The safety profile of AAV has been well-characterized in vivo studies, and the first gene therapy for patients with vision loss caused by Leber congenital amaurosis or retinitis pigmentosa was approved by the US Food and Drug Administration in 2017. This is an exciting era for investigators working on retina biology and treatments for blindness. In this chapter, we provide detailed methods for laboratory-scale production, purification, and characterization of AAV.

关键词

Adeno-associated virus; Packaging; Purification; Serotype; Titration