Skip navigation
Please use this identifier to cite or link to this item:
Full metadata record
DC FieldValueLanguage
dc.contributor.advisorMahmoud, Adel A.-
dc.contributor.authorMehta, Neil-
dc.description.abstractWhile genetic disorders are individually rare, many exist, and collectively these conditions affect millions of people. The lack of available treatments and cures for these conditions has resulted in hundreds of millions of dollars spent in caring for these patients each year globally. Gene therapy techniques were a first-pass attempt at curing these diseases; clinical trials for gene therapy increased dramatically throughout the 1990s into the 2000s. Gene therapy methods, however, were risky due to 1) adverse effects genetic vectors used had on patients and 2) a lack of knowledge of the human genome and the adverse effects foreign DNA expressed in humans might have. Current advances to both viral and non-viral genetic vectors have reduced immunogenicity and increased delivered DNA stability in host tissues. Next generation sequencing has enabled rapid, inexpensive, and accurate whole-genome sequencing that is very useful for not only learning more about the human genome and genetic disorders but also for providing patients better and more targeted care in the clinic. The development of CRISPR/Cas9 genome editing methodologies have revolutionized the possibly surrounding genome therapies in humans. These dramatic improvements in molecular biology, genomics, and bioengineering research all point to a revolution in modern medicine.en_US
dc.format.extent56 pages*
dc.titleAdvances in Molecular Biology in Progression Towards Human Genome Engineering Therapiesen_US
dc.typePrinceton University Senior Theses-
pu.departmentMolecular Biologyen_US
Appears in Collections:Molecular Biology, 1954-2023

Files in This Item:
File SizeFormat 
Mehta_senior_thesis_corrected.pdf775.93 kBAdobe PDF    Request a copy

Items in Dataspace are protected by copyright, with all rights reserved, unless otherwise indicated.