Genome Engineering Goes Viral: Repurposing of Adeno-associated Viral Vectors for CRISPR-mediated in Vivo Genome Engineering
One of the major challenges facing medicine and drug discovery is the large number of genetic diseases caused by inherited mutations leading to a toxic gain-of-function, or loss-of-function of the disease protein. Microbiology offered a new glimpse of hope to address those disorders with the adaptat...
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| Format: | Others |
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eScholarship@UMMS
2020
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| Online Access: | https://escholarship.umassmed.edu/gsbs_diss/1114 https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=2120&context=gsbs_diss |