CRISPR/Cas9 genome editing to treat sickle cell disease
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022.
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Brac University
2023
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| 在線閱讀: | http://hdl.handle.net/10361/19355 |
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10361-193552023-08-08T21:02:09Z CRISPR/Cas9 genome editing to treat sickle cell disease Ullah, Mohammad Islam, Farzana Department of Pharmacy, Brac University SCD CRISPR/Cas9 Hematopoietic stem/progenitor cells Fetal hemoglobin Gene editing tools Genetic engineering--Research Gene therapy--Research Biotechnology This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022. Cataloged from PDF version of thesis. Includes bibliographical references (pages 49-54). The most prevalent inherited blood condition, Sickle Cell Disease (SCD) causes excruciating pain, permanent organ damage including liver, heart, and kidney also causes an untimely death. There is currently a dearth of treatment options for SCD. In order to lessen the severity of acute problems, only four medications have been licensed by the Food and Drug Administration (FDA). Hematopoietic stem cell transplantation, is the curative treatment for SCD, that is related to matched donor. Recent and significant advancements in genome editing techniques have shown promise as a curative option, with the potential to fix the mutation in patient-derived Hematopoietic Stem/Progenitor Cells (HSPCs), to increase Fetal Hemoglobin (HbF) expression to avoid sickling of Red Blood Cells (RBCs), and generate corrected induced Pluripotent Stem Cells (iPSCs). CRISPR/Cas9, which was discovered relatively recently, has not only changed the face of genome engineering, but also made it possible to use these ideas in the clinic. In this review, we provide an overview of CRISPR/Cas9 applications in genome engineering and discuss the advantages, disadvantages, how it is superior to other gene editing technologies such as Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs) and potential of this technology as a treatment for SCD. Mohammad Ullah B. Pharmacy 2023-08-08T05:38:57Z 2023-08-08T05:38:57Z 2022 2022-11 Thesis ID 18346005 http://hdl.handle.net/10361/19355 en Brac University theses are protected by copyright. They may be viewed from this source for any purpose, but reproduction or distribution in any format is prohibited without written permission. 54 pages application/pdf Brac University |
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Brac University |
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Institutional Repository |
| language |
English |
| topic |
SCD CRISPR/Cas9 Hematopoietic stem/progenitor cells Fetal hemoglobin Gene editing tools Genetic engineering--Research Gene therapy--Research Biotechnology |
| spellingShingle |
SCD CRISPR/Cas9 Hematopoietic stem/progenitor cells Fetal hemoglobin Gene editing tools Genetic engineering--Research Gene therapy--Research Biotechnology Ullah, Mohammad CRISPR/Cas9 genome editing to treat sickle cell disease |
| description |
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022. |
| author2 |
Islam, Farzana |
| author_facet |
Islam, Farzana Ullah, Mohammad |
| format |
Thesis |
| author |
Ullah, Mohammad |
| author_sort |
Ullah, Mohammad |
| title |
CRISPR/Cas9 genome editing to treat sickle cell disease |
| title_short |
CRISPR/Cas9 genome editing to treat sickle cell disease |
| title_full |
CRISPR/Cas9 genome editing to treat sickle cell disease |
| title_fullStr |
CRISPR/Cas9 genome editing to treat sickle cell disease |
| title_full_unstemmed |
CRISPR/Cas9 genome editing to treat sickle cell disease |
| title_sort |
crispr/cas9 genome editing to treat sickle cell disease |
| publisher |
Brac University |
| publishDate |
2023 |
| url |
http://hdl.handle.net/10361/19355 |
| work_keys_str_mv |
AT ullahmohammad crisprcas9genomeeditingtotreatsicklecelldisease |
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1814309217512718336 |