Review on gene replacement therapy for spinal muscular atrophy
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2023.
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| Idioma: | English |
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Brac University
2023
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| Acesso em linha: | http://hdl.handle.net/10361/21242 |
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10361-21242 |
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10361-212422023-09-25T21:06:28Z Review on gene replacement therapy for spinal muscular atrophy Jannat, Nurani Haque, MD. Aminul Department of Pharmacy, Brac University Spinal muscular atrophy type I Gene therapy Motor milestone Genetic transformation Gene therapy This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2023. Cataloged from PDF version of thesis. Includes bibliographical references (pages 33-38). Spinal muscular atrophy (SMA) is initiated by the deletion or malfunction of the gene that encodes survival motor neuron 1 (SMN1) and this leads to muscular atrophy. It is one of the most prevalent hereditary that causes infant mortality. Eventually, various genetic interventions and approaches, including gene replacement, non-gene replacement, and gene editing have been explored up to this point as potential treatment options for spinal muscular atrophy and other neuromuscular illnesses. Whereas gene replacement therapy is most often claimed to treat infants and toddlers with SMA type I. When treating the underlying cause of this disease, researchers have discovered the greatest benefit to motor neurons early on. Here, I have reviewed the enormous advances made in genetically mediated therapeutics for SMA, with an emphasis on gene replacement therapy and the outcome of this therapy was found to be more efficacious than other treatments in terms of motor millstone result. Nurani Jannat B. Pharmacy 2023-09-25T07:31:15Z 2023-09-25T07:31:15Z 2023 2023-03 Thesis ID 18346010 http://hdl.handle.net/10361/21242 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. 38 pages application/pdf Brac University |
| institution |
Brac University |
| collection |
Institutional Repository |
| language |
English |
| topic |
Spinal muscular atrophy type I Gene therapy Motor milestone Genetic transformation Gene therapy |
| spellingShingle |
Spinal muscular atrophy type I Gene therapy Motor milestone Genetic transformation Gene therapy Jannat, Nurani Review on gene replacement therapy for spinal muscular atrophy |
| description |
This thesis is submitted in partial fulfillment of the requirements for the degree of Bachelor of Pharmacy, 2023. |
| author2 |
Haque, MD. Aminul |
| author_facet |
Haque, MD. Aminul Jannat, Nurani |
| format |
Thesis |
| author |
Jannat, Nurani |
| author_sort |
Jannat, Nurani |
| title |
Review on gene replacement therapy for spinal muscular atrophy |
| title_short |
Review on gene replacement therapy for spinal muscular atrophy |
| title_full |
Review on gene replacement therapy for spinal muscular atrophy |
| title_fullStr |
Review on gene replacement therapy for spinal muscular atrophy |
| title_full_unstemmed |
Review on gene replacement therapy for spinal muscular atrophy |
| title_sort |
review on gene replacement therapy for spinal muscular atrophy |
| publisher |
Brac University |
| publishDate |
2023 |
| url |
http://hdl.handle.net/10361/21242 |
| work_keys_str_mv |
AT jannatnurani reviewongenereplacementtherapyforspinalmuscularatrophy |
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1814308163884679168 |