Development of RNA interference-based therapy for rare genetic diseases
In December 2022, the Indonesian Ministry of Health projected that rare diseases might affect 8–10% of the population, equating to approximately 27 million people. These diseases, often congenital, are linked to genetic inheritance or mutations, leading to structural or functional defects. Despite...
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| Format: | Article |
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Indonesian Society for Biochemistry and Molecular Biology
2024-08-01
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| Series: | Acta Biochimica Indonesiana |
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| Online Access: | https://pbbmi.org/newjurnal/index.php/actabioina/article/view/171 |
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| author | Milya Urfa Ahmad Syarifah Dewi |
| author_facet | Milya Urfa Ahmad Syarifah Dewi |
| author_sort | Milya Urfa Ahmad |
| collection | DOAJ |
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In December 2022, the Indonesian Ministry of Health projected that rare diseases might affect 8–10% of the population, equating to approximately 27 million people. These diseases, often congenital, are linked to genetic inheritance or mutations, leading to structural or functional defects. Despite advancements in diagnostic and treatment methods, many rare diseases remain challenging for healthcare practitioners. RNA interference (RNAi) presents a promising therapeutic approach by enabling the selective inhibition of genes responsible for genetic disorders. RNAi employs small RNA molecules, such as small interfering RNA (siRNA) and microRNA (miRNA), to bind specific mRNA molecules and prevent their translation into proteins. Current research showed that RNAi-based therapies have the potential to treat various genetic diseases, including acute hepatic porphyria (AHP) and primary hyperoxaluria type 1 (PH1). However, the mechanisms of RNAi in hereditary disorders like AHP and PH1 require further documentation. RNAi offers several advantages, including gene-specific targeting, versatility in treating diverse genetic disorders, and scalability for mass production. Nonetheless, challenges remain, such as side effects, difficulties in targeting specific cells, and high development cost. Despite these obstacles, RNAi-based therapy holds significant potential for revolutionize the treatment of genetic disorders.
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| format | Article |
| id | doaj-art-1c548d3f7def4a518e01df474a9b74f6 |
| institution | Kabale University |
| issn | 2654-6108 2654-3222 |
| language | English |
| publishDate | 2024-08-01 |
| publisher | Indonesian Society for Biochemistry and Molecular Biology |
| record_format | Article |
| series | Acta Biochimica Indonesiana |
| spelling | doaj-art-1c548d3f7def4a518e01df474a9b74f62025-02-08T03:04:46ZengIndonesian Society for Biochemistry and Molecular BiologyActa Biochimica Indonesiana2654-61082654-32222024-08-017110.32889/actabioina.171Development of RNA interference-based therapy for rare genetic diseasesMilya Urfa Ahmad0Syarifah Dewi1Master’s Programme in Biomedical Sciences, Faculty of Medicine, Universitas IndonesiaDepartment of Biochemistry and Molecular Biology, Faculty of Medicine Universitas Indonesia, Jakarta, Indonesia In December 2022, the Indonesian Ministry of Health projected that rare diseases might affect 8–10% of the population, equating to approximately 27 million people. These diseases, often congenital, are linked to genetic inheritance or mutations, leading to structural or functional defects. Despite advancements in diagnostic and treatment methods, many rare diseases remain challenging for healthcare practitioners. RNA interference (RNAi) presents a promising therapeutic approach by enabling the selective inhibition of genes responsible for genetic disorders. RNAi employs small RNA molecules, such as small interfering RNA (siRNA) and microRNA (miRNA), to bind specific mRNA molecules and prevent their translation into proteins. Current research showed that RNAi-based therapies have the potential to treat various genetic diseases, including acute hepatic porphyria (AHP) and primary hyperoxaluria type 1 (PH1). However, the mechanisms of RNAi in hereditary disorders like AHP and PH1 require further documentation. RNAi offers several advantages, including gene-specific targeting, versatility in treating diverse genetic disorders, and scalability for mass production. Nonetheless, challenges remain, such as side effects, difficulties in targeting specific cells, and high development cost. Despite these obstacles, RNAi-based therapy holds significant potential for revolutionize the treatment of genetic disorders. https://pbbmi.org/newjurnal/index.php/actabioina/article/view/171RNA interferenceRNA therapygenetic diseaseacute hepatic porphyriaprimary hyperoxaluria type 1 |
| spellingShingle | Milya Urfa Ahmad Syarifah Dewi Development of RNA interference-based therapy for rare genetic diseases Acta Biochimica Indonesiana RNA interference RNA therapy genetic disease acute hepatic porphyria primary hyperoxaluria type 1 |
| title | Development of RNA interference-based therapy for rare genetic diseases |
| title_full | Development of RNA interference-based therapy for rare genetic diseases |
| title_fullStr | Development of RNA interference-based therapy for rare genetic diseases |
| title_full_unstemmed | Development of RNA interference-based therapy for rare genetic diseases |
| title_short | Development of RNA interference-based therapy for rare genetic diseases |
| title_sort | development of rna interference based therapy for rare genetic diseases |
| topic | RNA interference RNA therapy genetic disease acute hepatic porphyria primary hyperoxaluria type 1 |
| url | https://pbbmi.org/newjurnal/index.php/actabioina/article/view/171 |
| work_keys_str_mv | AT milyaurfaahmad developmentofrnainterferencebasedtherapyforraregeneticdiseases AT syarifahdewi developmentofrnainterferencebasedtherapyforraregeneticdiseases |