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Keywords: Antisense oligonucleotide
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Journal Articles
Dis Model Mech (2022) 15 (7): dmm049284.
Published: 25 July 2022
... was then used to test the therapeutic efficacy of Dnm2 targeting. Dnm2 reduction by antisense oligonucleotides blocked or postponed disease development, and resulted in a significant dose-dependent improvement outside the expected disease progression in untreated Mtm1 −/y mice. This provides an example...
Includes: Supplementary data
Journal Articles
Dis Model Mech (2020) 13 (2): dmm040832.
Published: 4 November 2019
... ) gene. The most common SGCG mutation is a single nucleotide deletion from a stretch of five thymine residues in SGCG exon 6 (521ΔT). This founder mutation disrupts the transcript reading frame, abolishing protein expression. An antisense oligonucleotide exon-skipping method to reframe the human 521ΔT...
Includes: Supplementary data