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dc.contributor.advisor Bao, Gang
dc.creatorLi, Ang
dc.date.accessioned 2020-08-18T12:55:19Z
dc.date.available 2021-02-01T06:01:12Z
dc.date.created 2020-08
dc.date.issued 2020-08-12
dc.date.submitted August 2020
dc.identifier.citation Li, Ang. "Development and Characterization of Viral-Based Gene Editing In Vivo." (2020) Diss., Rice University. https://hdl.handle.net/1911/109234.
dc.identifier.urihttps://hdl.handle.net/1911/109234
dc.description.abstract Adeno-Associated Viral (AAV) vectors packaging the CRISPR/Cas9 system (AAVCRISPR) can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy because of their ability to achieve sustained expression from largely non-integrating episomal genomes. However, for genome editing applications, permanent expression of non-human proteins such as the bacterially-derived Cas9 nuclease is undesirable. Recent studies indicate a high prevalence of neutralizing antibodies and T-cells specific to the commonly used Cas9 orthologs from Streptococcus pyogenes (SpCas9) and Staphylococcus aureus (SaCas9) in humans. Additionally, persistent expression of CRISPR/Cas9 has the potential to increase the chances of off-target cutting. There is a need for efficient genome editing in vivo, with controlled transient expression of CRISPR/Cas9. The topic of my thesis covers the development of a self-deleting AAVCRISPR system that introduces insertion and deletion mutations into AAV episomes, understanding the effects AAV-CRISPR editing in vivo in a Cas9 immunized mouse model, and the characterization of AAV integrations into the genome in the context of CRISPR-based gene editing.
dc.format.mimetype application/pdf
dc.language.iso eng
dc.subjectGene Editing
Adeno-Associated Virus
Gene Therapy
CRISPR/Cas9
dc.title Development and Characterization of Viral-Based Gene Editing In Vivo
dc.type Thesis
dc.date.updated 2020-08-18T12:55:20Z
dc.type.material Text
thesis.degree.department Bioengineering
thesis.degree.discipline Engineering
thesis.degree.grantor Rice University
thesis.degree.level Doctoral
thesis.degree.name Doctor of Philosophy
dc.embargo.terms 2021-02-01


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