Gene therapy for rare genetic disorders
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022.
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| פורמט: | Thesis |
| שפה: | English |
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Brac University
2024
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| גישה מקוונת: | http://hdl.handle.net/10361/22661 |
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10361-22661 |
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10361-226612024-04-24T21:02:36Z Gene therapy for rare genetic disorders Tanjum, Atia Sayka Khan, Tanisha Tabassum School of Pharmacy, Brac University Gene therapy Vectors Rare genetic disorders Clinical trial Gene therapy. Genetic disorders. Genetic disorders--Treatment. 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 54-58). Genetic disorders are widespread. However, many of these genetic illnesses have minimal information, making therapy difficult. Gene therapy has emerged as a major breakthrough in the field of medical science to treat diseases resulting from defective genes. The FDA has received over 900 gene therapies for hereditary disorders. Rare genetic illnesses are life threatening and impact 5 per 10000 people worldwide. Researchers are conducting clinical trials to investigate treatments for rare genetic disorders despite a paucity of knowledge. With sufficient clinical trial data showing signs of improvement among the patients and safety of gene delivery systems, gene therapy can be the most efficient treatment strategy for rare genetic disorders. In this review paper, a number of rare genetic disorders and the prospects of gene therapy in the treatment of those disorders were discussed. Atia Tanjum B. Pharmacy 2024-04-24T04:54:01Z 2024-04-24T04:54:01Z 2022 2022-10 Thesis ID: 15346034 http://hdl.handle.net/10361/22661 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. 58 pages application/pdf Brac University |
| institution |
Brac University |
| collection |
Institutional Repository |
| language |
English |
| topic |
Gene therapy Vectors Rare genetic disorders Clinical trial Gene therapy. Genetic disorders. Genetic disorders--Treatment. |
| spellingShingle |
Gene therapy Vectors Rare genetic disorders Clinical trial Gene therapy. Genetic disorders. Genetic disorders--Treatment. Tanjum, Atia Gene therapy for rare genetic disorders |
| description |
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2022. |
| author2 |
Sayka Khan, Tanisha Tabassum |
| author_facet |
Sayka Khan, Tanisha Tabassum Tanjum, Atia |
| format |
Thesis |
| author |
Tanjum, Atia |
| author_sort |
Tanjum, Atia |
| title |
Gene therapy for rare genetic disorders |
| title_short |
Gene therapy for rare genetic disorders |
| title_full |
Gene therapy for rare genetic disorders |
| title_fullStr |
Gene therapy for rare genetic disorders |
| title_full_unstemmed |
Gene therapy for rare genetic disorders |
| title_sort |
gene therapy for rare genetic disorders |
| publisher |
Brac University |
| publishDate |
2024 |
| url |
http://hdl.handle.net/10361/22661 |
| work_keys_str_mv |
AT tanjumatia genetherapyforraregeneticdisorders |
| _version_ |
1814308169635069952 |