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Hope on the Horizon: New and Future Therapies for Sickle Cell Disease
This article provides an overview of conventional, new, and future treatment options for sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current treatments include hydroxyurea, a conventional SCD treatment that increases the levels of fetal hemoglobin, and new t...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2023
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10488840/ https://www.ncbi.nlm.nih.gov/pubmed/37685759 http://dx.doi.org/10.3390/jcm12175692 |
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author | Kuriri, Fahd A. |
author_facet | Kuriri, Fahd A. |
author_sort | Kuriri, Fahd A. |
collection | PubMed |
description | This article provides an overview of conventional, new, and future treatment options for sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current treatments include hydroxyurea, a conventional SCD treatment that increases the levels of fetal hemoglobin, and new treatments such as voxelotor, a recently approved SCD treatment that selectively binds hemoglobin, preventing formation of sickled red blood cells. In addition to discussing the mechanisms of action of current SCD treatments, potential side effects are also discussed, highlighting the need for new treatments that can address the limitations of current treatments and improve the quality of life for people with SCD. Future treatments, such as gene therapy, are also explored as promising treatment options for SCD patients. |
format | Online Article Text |
id | pubmed-10488840 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-104888402023-09-09 Hope on the Horizon: New and Future Therapies for Sickle Cell Disease Kuriri, Fahd A. J Clin Med Review This article provides an overview of conventional, new, and future treatment options for sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current treatments include hydroxyurea, a conventional SCD treatment that increases the levels of fetal hemoglobin, and new treatments such as voxelotor, a recently approved SCD treatment that selectively binds hemoglobin, preventing formation of sickled red blood cells. In addition to discussing the mechanisms of action of current SCD treatments, potential side effects are also discussed, highlighting the need for new treatments that can address the limitations of current treatments and improve the quality of life for people with SCD. Future treatments, such as gene therapy, are also explored as promising treatment options for SCD patients. MDPI 2023-09-01 /pmc/articles/PMC10488840/ /pubmed/37685759 http://dx.doi.org/10.3390/jcm12175692 Text en © 2023 by the author. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Kuriri, Fahd A. Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title | Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title_full | Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title_fullStr | Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title_full_unstemmed | Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title_short | Hope on the Horizon: New and Future Therapies for Sickle Cell Disease |
title_sort | hope on the horizon: new and future therapies for sickle cell disease |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10488840/ https://www.ncbi.nlm.nih.gov/pubmed/37685759 http://dx.doi.org/10.3390/jcm12175692 |
work_keys_str_mv | AT kuririfahda hopeonthehorizonnewandfuturetherapiesforsicklecelldisease |