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Towards Therapeutic Genome Editing: From Reducing Population Disease Risk to Curing Ultra-Rare Genetic Disorders Madelynn N Whittaker
- Format:
- Book
- Thesis/Dissertation
- Author/Creator:
- Whittaker, Madelynn N., author.
- Language:
- English
- Subjects (All):
- Bioengineering.
- Genetics.
- Molecular biology.
- Pathology.
- 0202.
- 0369.
- 0307.
- 0571.
- Local Subjects:
- Bioengineering.
- Genetics.
- Molecular biology.
- Pathology.
- 0202.
- 0369.
- 0307.
- 0571.
- Physical Description:
- 1 electronic resource (142 pages)
- Contained In:
- Dissertations Abstracts International 86-12B
- Place of Publication:
- Ann Arbor : ProQuest Dissertations and Theses, 2025
- Language Note:
- English
- Summary:
- CRISPR-Cas9-derived genome editing technologies-base editing, prime editing, and epigenome editing-offer the ability to modify the genome with remarkable specificity and efficiency, revolutionizing the potential for precision medicine. However, each technology faces distinct limitations that hinder its widespread clinical adoption. This thesis aims to bridge the gap between these gene editing tools and their promise for clinical translation. Specifically, this work focuses on improving the therapeutic potential of these technologies by examining editing efficiencies over diverse genetic loci, enhancing the specificity profiles of gene editing strategies, and developing streamlined approaches to identify lead editing configurations. This research advances the ongoing effort to establish gene editing as a safe and viable therapeutic strategy for a broad spectrum of genetic disorders. By addressing current limitations, I explore gene editing as a tool for treating genetic diseases at both the population and individual levels
- Notes:
- Source: Dissertations Abstracts International, Volume: 86-12, Section: B.
- Includes supplementary digital materials
- Advisors: Musunuru, Kiran Committee members: Ahrens-Nicklas, Rebecca; Grunewald, Julian; Raj, Arjun
- Ph.D. University of Pennsylvania 2025
- Local Notes:
- School code: 0175
- ISBN:
- 9798280759251
- Access Restriction:
- Restricted for use by site license
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