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Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea

Hum Gene Ther. 2020; 
Amy Frederick, Jennifer Sullivan, Lin Liu, Matthew Adamowicz, Michael Lukason, Jasmine Raymer, Zhengyu Luo, Xiaoying Jin, Kollu Nageswara Rao, Catherine O'Riordan
Products/Services Used Details Operation
GenParts™ DNA Fragments … Deamidation variants N57D and G58D are based on pim45BD-cap2, an AAV helper plasmid that contains both rep and cap sequences from AAV2. Fragments containing the designated mutations were synthesized (Genscript) and subcloned into pim45BD-cap2 … Get A Quote

摘要

Adeno-associated viral (AAV) vectors represent an ideal vehicle for human gene transfer. One advantage to the AAV vector system is the availability of multiple naturally occurring serotypes that provide selective tropisms for various target cells. Strategies to enhance the properties of the natural AAV isolates have been developed and can be divided into two approaches, rational design or directed evolution. The rational design approach utilizes knowledge of AAV capsids to make targeted changes to the capsid to alter transduction efficiency or specificity, while the directed evolution approach does not require knowledge of capsid structure and includes random mutagenesis, capsid shuffling, or random peptide in... More

关键词

AAV capsid engineering, AAV capsid posttranslational modifications, AAV capsid variants, ocular gene therapy