Research from D.O. Ott Research Institute of Obstetrics Yields New Data on Antisense Technology (Development of 2'-O-Methyl and LNA Antisense Oligonucleotides for SMN2 Splicing Correction in SMA Cells).
In: Gene Therapy Weekly, 2023-12-04, S. 100-100
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Zugriff:
A study conducted by the D.O. Ott Research Institute of Obstetrics has explored the use of antisense technology in treating spinal muscular atrophy (SMA), a neurodegenerative disease caused by mutations in the SMN1 gene. The researchers designed antisense oligonucleotides (AONs) to target specific regions in the SMN2 gene and tested their efficacy in SMA-cultured fibroblasts. The study found that certain AONs led to a significant increase in full-length SMN transcripts and nuclear gems, reaching levels comparable to those found in healthy cells. The researchers also observed a synergistic effect when combining different AONs. The impact of the oligonucleotides was found to be influenced by their length, sequence, and pattern of modification. This research provides valuable insights for the development of more effective and affordable therapies for SMA. [Extracted from the article]
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Research from D.O. Ott Research Institute of Obstetrics Yields New Data on Antisense Technology (Development of 2'-O-Methyl and LNA Antisense Oligonucleotides for SMN2 Splicing Correction in SMA Cells).
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Zeitschrift: | Gene Therapy Weekly, 2023-12-04, S. 100-100 |
Veröffentlichung: | 2023 |
Medientyp: | serialPeriodical |
ISSN: | 1078-2842 (print) |
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