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Simon Alexander Krooss on Muck Rack

Simon Alexander Krooss

As seen in: PLOS ONE, Cell Press
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Articles

Ex Vivo/In vivo Gene Editing in Hepatocytes Using “All-in-One” CRISPR-Adeno-Associated Virus Vectors with a Self-Linearizing Repair Template

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By Zhen Dai, Florian Schmidt, Simon Alexander Krooss, Alice Rovai
| Cell Press Verified
Highlights • Single AAV vector mediates efficient large fragment replacement in vivo and ex vivo • Fah-corrected iHeps repopulate the liver of recipient mice • Self-linearizing donor template enhances integration rate • AAV2 and AAV8 reveal differences in packaging the oversized AIO-SL vector genome Summary Adeno-associated virus (AAV)-based vectors are considered efficient and safe gene delivery systems in gene therapy.
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