Non-deletional alpha thalassaemia: a review of emerging therapy
More than seventy non-deletional α-thalassaemia mutations have been reported and they usually exhibit more severe clinical presentations compared to their deletional counterparts. Conventional treatment aims to manage the symptoms of the disease through red blood cell transfusion, but this has its...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Universiti Putra Malaysia
2021
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| Online Access: | http://psasir.upm.edu.my/id/eprint/94367/ http://psasir.upm.edu.my/id/eprint/94367/1/Non-Deletional%20Alpha%20Thalassaemia_A%20Review%20of%20Emerging%20Therapy.pdf |
| _version_ | 1848861977993019392 |
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| author | Khairil Wahidin, Nurfhaezah Lai, Mei I. |
| author_facet | Khairil Wahidin, Nurfhaezah Lai, Mei I. |
| author_sort | Khairil Wahidin, Nurfhaezah |
| building | UPM Institutional Repository |
| collection | Online Access |
| description | More than seventy non-deletional α-thalassaemia mutations have been reported and they usually exhibit more severe clinical presentations compared to their deletional counterparts. Conventional treatment aims to manage the
symptoms of the disease through red blood cell transfusion, but this has its own set of complications. For the time
being, the only cure for thalassaemia is bone marrow transplantation, hence, it is a priority to explore other potential
treatment methods. Novel gene editing methods could potentially be a long-term treatment option for this single
gene disorder. This manuscript provides an overview of recent breakthroughs in non-deletional α-thalassaemia treatment, including intrauterine transfusion, cord blood transplantation, gene therapy, and several genome modification
techniques, to contribute to the overall knowledge in not only ameliorating the condition of α-thalassaemia, but also
to find a cure. |
| first_indexed | 2025-11-15T13:09:42Z |
| format | Article |
| id | upm-94367 |
| institution | Universiti Putra Malaysia |
| institution_category | Local University |
| language | English |
| last_indexed | 2025-11-15T13:09:42Z |
| publishDate | 2021 |
| publisher | Universiti Putra Malaysia |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | upm-943672023-04-04T02:26:01Z http://psasir.upm.edu.my/id/eprint/94367/ Non-deletional alpha thalassaemia: a review of emerging therapy Khairil Wahidin, Nurfhaezah Lai, Mei I. More than seventy non-deletional α-thalassaemia mutations have been reported and they usually exhibit more severe clinical presentations compared to their deletional counterparts. Conventional treatment aims to manage the symptoms of the disease through red blood cell transfusion, but this has its own set of complications. For the time being, the only cure for thalassaemia is bone marrow transplantation, hence, it is a priority to explore other potential treatment methods. Novel gene editing methods could potentially be a long-term treatment option for this single gene disorder. This manuscript provides an overview of recent breakthroughs in non-deletional α-thalassaemia treatment, including intrauterine transfusion, cord blood transplantation, gene therapy, and several genome modification techniques, to contribute to the overall knowledge in not only ameliorating the condition of α-thalassaemia, but also to find a cure. Universiti Putra Malaysia 2021-12 Article PeerReviewed text en http://psasir.upm.edu.my/id/eprint/94367/1/Non-Deletional%20Alpha%20Thalassaemia_A%20Review%20of%20Emerging%20Therapy.pdf Khairil Wahidin, Nurfhaezah and Lai, Mei I. (2021) Non-deletional alpha thalassaemia: a review of emerging therapy. Malaysian Journal of Medicine and Health Sciences, 17 (suppl. 10). art. no. 11. 72 - 84. ISSN 2636-9346 https://medic.upm.edu.my/upload/dokumen/2021120708205111_MJMHS_0783.pdf |
| spellingShingle | Khairil Wahidin, Nurfhaezah Lai, Mei I. Non-deletional alpha thalassaemia: a review of emerging therapy |
| title | Non-deletional alpha thalassaemia: a review of emerging therapy |
| title_full | Non-deletional alpha thalassaemia: a review of emerging therapy |
| title_fullStr | Non-deletional alpha thalassaemia: a review of emerging therapy |
| title_full_unstemmed | Non-deletional alpha thalassaemia: a review of emerging therapy |
| title_short | Non-deletional alpha thalassaemia: a review of emerging therapy |
| title_sort | non-deletional alpha thalassaemia: a review of emerging therapy |
| url | http://psasir.upm.edu.my/id/eprint/94367/ http://psasir.upm.edu.my/id/eprint/94367/ http://psasir.upm.edu.my/id/eprint/94367/1/Non-Deletional%20Alpha%20Thalassaemia_A%20Review%20of%20Emerging%20Therapy.pdf |