Date of Award

2022

Document Type

Restricted Access Thesis

Degree Name

MS in Physician Assistant Studies (PA)

Department

Physician Assistant Studies

First Advisor

Ryane Lester, PA-C

Abstract

Background: Sickle cell disease is a genetic disorder that causes several morbidities and increased mortality. The only definitive treatment currently available is an allogenic bone marrow transplant with strict criteria for participation. CRISPR gene editing could be a solution to fixing the gene itself to create healthy hemoglobin and be a more inclusive curative treatment option.

Purpose: Can CRISPR gene editing technology be used to definitively treat sickle cell disease? Methods: A comprehensive literature review was conducted used Trip Medical Database using the search terms ‘sickle cell disease,’ ‘gene editing therapy,’ ‘curative or definitive treatments.’ Inclusion criteria included modern studies relating to the history and progression of finding a definitive treatment for sickle cell disease by altering a person’s DNA.

Conclusions: There are two methods highlighted in this report that have shown potential to be curative treatment options for sickle cell disease; using a lentiviral vector to correct the mutated nucleotide that causes the disease itself and using electroporation to since BCL11A to induce the production of more fetal hemoglobin. Both methods are in the beginning phases of being tested and proven successful in human patients. More time and participants are needed to investigate the long term effects and sustainability of the genetic modifications.

Identifier

SC 11.PAS.2022.Bartosh.A

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