Strategies for Expressing Transgenes that Exceed the Packaging Capacity of AAVs
Description
This template was adapted from the original submission. Edits were made to enhance scientific accuracy, optimal usability and/or to meet industry-leading design standards for science communication.
Adeno-associated viral (AAV) vectors are popular gene therapy tools, but their genome packaging limit is ~4.7 kb. To exceed this size, transgenes are split between two AAVs and the two halves joined by use of: transplicing via a splice site donor and acceptor, homologous recombination (HR) mediated by homologous arms (HA); or a hybrid of these two approaches. CRISPR-mediate HR donor templates can also be split and are fully incorporated following sequential HR.
Acknowledgements
References
Kyle Chamberlain. (2016) Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids. Human Gene Therapy Methods. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4761816/
Rasmus O. Bak. (2017) CRISPR-mediated Integration of Large Gene Cassettes using AAV Donor Vectors. Cell Reports. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5568673/
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