Exact(3)
So, we follow [38] to consider a truncated functional.
So we follow [23] to consider a truncated functional of (J u)).
In the presence of NMD, less of the truncated functional protein will be produced, which can exacerbate disease symptoms.
Similar(57)
Our results demonstrate that truncated but functional dystrophin protein expression improved the cellular phenotype of DMD myotubes.
Exon skipping is a therapeutic approach that uses antisense oligonucleotides (AOs) to modulate splicing and restore the reading frame, leading to truncated, yet functional protein expression.
Antisense oligonucleotides (AOs) have previously been used to skip additional exons that border the deletions such that the reading frame is restored and internally truncated, but functional, dystrophin expressed.
Modulation of pre-mRNA splicing with antisense oligonucleotides (AOs) to restore the reading frame has been demonstrated in vitro and in vivo, such that truncated but functional dystrophin is expressed.
Exon-skipping phosphorodiamidate morpholino oligomers (PMOs) direct alternative splicing of dystrophin pre-mRNA, restoring the mRNA reading frame and enabling translation of an internally truncated yet functional dystrophin protein.
Eteplirsen, an investigational PMO designed to enable production of internally truncated yet functional dystrophin in boys amenable to exon 51-skipping, was evaluated in previously conducted clinical studies (33, 28, and 201), and is currently being evaluated in ongoing clinical trials.
Antisense oligonucleotide-mediated exon skipping is able to correct out-of-frame mutations in Duchenne muscular dystrophy and restore truncated yet functional dystrophins.
The remaining four (OsRac1 4) are type II Rac/Rop proteins that carry a truncated but functional post-translational modification motif.
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