The potential role of miRNA therapies in spinal muscle atrophy
Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of full-length survival motor neuron (SMN) protein due to the loss of the survival motor neuron 1 (SMN1) gene and inefficient splicing of the survival motor neuron 2 (SMN2) gene, which mostly affects alpha motor neuron...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Elsevier BV
2021
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| Online Access: | http://psasir.upm.edu.my/id/eprint/95562/ http://psasir.upm.edu.my/id/eprint/95562/1/The%20potential%20role%20of%20miRNA%20therapies%20in%20spinal%20muscle%20atrophy.pdf |
| _version_ | 1848862189647036416 |
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| author | Gandhi, Gayatri Abdullah, Syahrilnizam Foead, Agus Iwan Yeo, Wendy Wai Yeng |
| author_facet | Gandhi, Gayatri Abdullah, Syahrilnizam Foead, Agus Iwan Yeo, Wendy Wai Yeng |
| author_sort | Gandhi, Gayatri |
| building | UPM Institutional Repository |
| collection | Online Access |
| description | Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of full-length survival motor neuron (SMN) protein due to the loss of the survival motor neuron 1 (SMN1) gene and inefficient splicing of the survival motor neuron 2 (SMN2) gene, which mostly affects alpha motor neurons of the lower spinal cord. Despite the U.S. Food and Drug Administration (FDA) approved SMN-dependent therapies including Nusinersen, Zolgensma® and Evrysdi™, SMA is still a devastating disease as these existing expensive drugs may not be sufficient and thus, remains a need for additional therapies. The involvement of microRNAs (miRNAs) in SMA is expanding because miRNAs are important mediators of gene expression as each miRNA could target a number of genes. Hence, miRNA-based therapy could be utilized in treating this genetic disorder. However, the delivery of miRNAs into the target cells remains an obstacle in SMA, as there is no effective delivery system to date. This review highlights the potential strategies for intracellular miRNA delivery into target cells and current challenges in miRNA delivery. Furthermore, we provide the future prospects of miRNA-based therapeutic strategies in SMA. |
| first_indexed | 2025-11-15T13:13:04Z |
| format | Article |
| id | upm-95562 |
| institution | Universiti Putra Malaysia |
| institution_category | Local University |
| language | English |
| last_indexed | 2025-11-15T13:13:04Z |
| publishDate | 2021 |
| publisher | Elsevier BV |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | upm-955622022-08-02T07:12:09Z http://psasir.upm.edu.my/id/eprint/95562/ The potential role of miRNA therapies in spinal muscle atrophy Gandhi, Gayatri Abdullah, Syahrilnizam Foead, Agus Iwan Yeo, Wendy Wai Yeng Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of full-length survival motor neuron (SMN) protein due to the loss of the survival motor neuron 1 (SMN1) gene and inefficient splicing of the survival motor neuron 2 (SMN2) gene, which mostly affects alpha motor neurons of the lower spinal cord. Despite the U.S. Food and Drug Administration (FDA) approved SMN-dependent therapies including Nusinersen, Zolgensma® and Evrysdi™, SMA is still a devastating disease as these existing expensive drugs may not be sufficient and thus, remains a need for additional therapies. The involvement of microRNAs (miRNAs) in SMA is expanding because miRNAs are important mediators of gene expression as each miRNA could target a number of genes. Hence, miRNA-based therapy could be utilized in treating this genetic disorder. However, the delivery of miRNAs into the target cells remains an obstacle in SMA, as there is no effective delivery system to date. This review highlights the potential strategies for intracellular miRNA delivery into target cells and current challenges in miRNA delivery. Furthermore, we provide the future prospects of miRNA-based therapeutic strategies in SMA. Elsevier BV 2021-08 Article PeerReviewed text en http://psasir.upm.edu.my/id/eprint/95562/1/The%20potential%20role%20of%20miRNA%20therapies%20in%20spinal%20muscle%20atrophy.pdf Gandhi, Gayatri and Abdullah, Syahrilnizam and Foead, Agus Iwan and Yeo, Wendy Wai Yeng (2021) The potential role of miRNA therapies in spinal muscle atrophy. Journal of the Neurological Sciences, 427. pp. 117485-117495. ISSN 0022-510X; ESSN: 1878-5883 https://www.sciencedirect.com/science/article/pii/S0022510X21001799?via%3Dihub 10.1016/j.jns.2021.117485 |
| spellingShingle | Gandhi, Gayatri Abdullah, Syahrilnizam Foead, Agus Iwan Yeo, Wendy Wai Yeng The potential role of miRNA therapies in spinal muscle atrophy |
| title | The potential role of miRNA therapies in spinal muscle atrophy |
| title_full | The potential role of miRNA therapies in spinal muscle atrophy |
| title_fullStr | The potential role of miRNA therapies in spinal muscle atrophy |
| title_full_unstemmed | The potential role of miRNA therapies in spinal muscle atrophy |
| title_short | The potential role of miRNA therapies in spinal muscle atrophy |
| title_sort | potential role of mirna therapies in spinal muscle atrophy |
| url | http://psasir.upm.edu.my/id/eprint/95562/ http://psasir.upm.edu.my/id/eprint/95562/ http://psasir.upm.edu.my/id/eprint/95562/ http://psasir.upm.edu.my/id/eprint/95562/1/The%20potential%20role%20of%20miRNA%20therapies%20in%20spinal%20muscle%20atrophy.pdf |