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Non-deletional alpha thalassaemia: a review of emerging therapy
Nurfhaezah Khairil Wahidin1, Mei I Lai2.
More than seventy non-deletional α-thalassaemia mutations have been reported and they usually exhibit more se- vere 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 treat- ment, 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.
Affiliation:
- University Putra Malaysia, Malaysia, 43400 Serdang, Malaysia
- University Putra Malaysia, Malaysia, 43400 Serdang, Malaysia
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Indexation |
Indexed by |
MyJurnal (2021) |
H-Index
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3 |
Immediacy Index
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0.000 |
Rank |
0 |
Indexed by |
Scopus 2020 |
Impact Factor
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CiteScore (0.2) |
Rank |
Q4 (Medicine (all)) |
Additional Information |
SJR (0.144) |
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