Therapeutic genome and cellular engineering with advanced programmable molecular tools

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dc.contributor.advisorGao, Xue Sherryen_US
dc.creatorYuan, Qichenen_US
dc.date.accessioned2023-08-09T15:22:28Zen_US
dc.date.created2023-05en_US
dc.date.issued2023-04-17en_US
dc.date.submittedMay 2023en_US
dc.date.updated2023-08-09T15:22:28Zen_US
dc.description.abstractNucleic acid engineering is a group of technologies that can change the script of life, such as genome and transcriptome, enabling a better understanding of human genomics, and can be developed as genetic medicines to treat diseases. Currently, there are three major types of gene editing approaches, including nuclease editing, base editing, and prime editing. Yet, the therapeutic applications of those technologies are still facing unmet needs. Although gene editing strategies have been demonstrated for monogenic disorders, such as sickle cell anemia, cystic fibrosis, Huntington disease, and Duchenne muscular dystrophy, the genetic or cellular treatment of polygenic disorder that caused by combined dysfunctions of more than one gene, such as coronary heart disease, diabetes, cancer, and neurological diseases, still needs to be well developed. How to further advance those technologies, or to develop next-generation gene editing tools that can perfectly address the emerging challenges from real-life medical issues? To approach a solution of the previous question, I focused my research on six topics: 1) Enable multiplex precision genome engineering with minimal delivery size, 2) Expand the type and number of genetic perturbations, 3) Manipulate cellular endogenous biological mechanisms to advance the performance of molecular tools, 4) Engineer the key components of molecular tools with improved activity, 5) Demonstrate therapeutic genome engineering on disease-relevant genes, 6) Deliver the therapeutic genetic payload using virial and non-viral approaches.en_US
dc.embargo.lift2024-05-01en_US
dc.embargo.terms2024-05-01en_US
dc.format.mimetypeapplication/pdfen_US
dc.identifier.citationYuan, Qichen. "Therapeutic genome and cellular engineering with advanced programmable molecular tools." (2023) Diss., Rice University. <a href="https://hdl.handle.net/1911/115090">https://hdl.handle.net/1911/115090</a>.en_US
dc.identifier.urihttps://hdl.handle.net/1911/115090en_US
dc.language.isoengen_US
dc.rightsCopyright is held by the author, unless otherwise indicated. Permission to reuse, publish, or reproduce the work beyond the bounds of fair use or other exemptions to copyright law must be obtained from the copyright holder.en_US
dc.subjectGenome Engineeringen_US
dc.subjectTranscriptome Engineeringen_US
dc.subjectCellular Engineeringen_US
dc.subjectCRISPRen_US
dc.subjectGene Therapyen_US
dc.subjectCell Therapyen_US
dc.subjectDeliveryen_US
dc.subjectPolygenic Diseaseen_US
dc.titleTherapeutic genome and cellular engineering with advanced programmable molecular toolsen_US
dc.typeThesisen_US
dc.type.materialTexten_US
thesis.degree.departmentChemical and Biomolecular Engineeringen_US
thesis.degree.disciplineEngineeringen_US
thesis.degree.grantorRice Universityen_US
thesis.degree.levelDoctoralen_US
thesis.degree.nameDoctor of Philosophyen_US
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