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Thus, both ESCs and iPSCs effect functional corrections in mdx muscle.
This suggests that the degree of molecular corrections depends on the number of WT ESCs incorporated and confirms our previous observations indicating that a minimal incorporation of ESCs (less than 5%) is insufficient to effect histological and functional corrections in the mdx musculature.
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Goyenvalle, A. et al. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
Recent data demonstrate promising effects of combining a corrector with a potentiator — an agent that increases the r∆F508-CFTR Cl- channel function — although the level of functional correction by such complementary therapy in CF patients is still far from that of WT-CFTR.
Conclusion: Suspension of the vaginal vault to the proximal uterosacral ligaments combined with site-specific repair of endopelvic fascia defects provides excellent anatomic and functional correction of pelvic organ prolapse in most women.
This functional correction was associated with an increase in the number of pancreatic islets and with the recovery of the anatomical and numerical distribution of insulin- and glucagon-producing cells inside them.
https://doi.org/10.1007/s00439-017-1801-z. Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, et al. TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.
In order to evaluate the functional correction induced by exon-skipping, we first assessed the histopathology of the treated muscles.
Accordingly, we have reported that a decrease in K8 expression leads to functional correction of ΔF508-CFTR (Colas et al, 2012).
In addition, because of the complex post-translational pathway required by CFTR, mRNA levels may in fact not correlate with levels of protein or degree of functional correction.
Sweeney CL, Zou J, Choi U, Merling RK, Liu A, Bodansky A, et al. Targeted repair of CYBB in X-CGD iPSCs requires retention of intronic sequences for expression and functional correction.
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