Liberate
发表于 2025-3-28 18:40:06
https://doi.org/10.1007/978-3-322-88352-0 patient to myotubes by MyoD transduction using fluorescence-activated cell sorting (FACS). We subsequently designed antisense PMOs targeting identical regions of dog and human dystrophin exons 6 and 8 and administered them as a cocktail to the in vitro generated dog or human myotubes. In both cases
Costume
发表于 2025-3-28 19:42:39
https://doi.org/10.1007/978-3-663-05507-5tiated myoblasts. Herein, we describe an optimized methodology to prepare myoblasts differentiated from iPS cells by mRNA transfection of the CRISPR-Cas9 system to skip exon 45 in myoblasts, and evaluate the restored dystrophin by RT-PCR and Western blotting.
尊重
发表于 2025-3-29 00:29:01
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Initial
发表于 2025-3-29 05:23:41
https://doi.org/10.1007/978-3-658-10661-4pecific exon 51 or 53 targeting AONs on RNA, protein, histological, and functional levels. Therefore, the model can be used to optimize human specific AONs, e.g., by comparing dystrophin protein and exon skipping levels..Absolute quantification of exon skipping levels can be obtained by digital drop
cardiopulmonary
发表于 2025-3-29 10:38:42
Invention and Early History of Exon Skipping and Splice Modulationase. Defects in RNA splicing account for at least 10% of all genetic disorders, with the number expected to increase as more information is uncovered on the contribution of noncoding genomic regions to disease. Splice modulation through the use of antisense oligonucleotides (AOs) has emerged as a pr
etiquette
发表于 2025-3-29 15:09:25
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Nonthreatening
发表于 2025-3-29 16:39:20
Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophytein. Insufficient levels of SMN results in the loss of motor neurons, which causes muscle weakness, respiratory distress, and paralysis. A nearly identical gene (.) contains a C-to-T transition which excludes exon 7 from 90% of the mature mRNA transcripts, leading to unstable proteins which are tar
碎石头
发表于 2025-3-29 23:46:38
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Vo2-Max
发表于 2025-3-30 01:27:57
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泥沼
发表于 2025-3-30 07:52:34
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