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Drug Discovery Collection: This Editorial reviews problems and some solutions to the translational use of animal models in the rare neuromuscular diseases context.
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Here we discuss current efforts to design gene therapy strategies to alleviate ER stress on a disease context.
Cumulatively, these findings indicate that the pharmacological inhibition or activation of the Hippo pathway could be desirable depending on the disease context.
In summary, we demonstrated here that integration-free iPSCs can be efficiently and consistently obtained from non-invasively acquired human EMCs and UCs, even in a defective disease context (i.e. type II DM) (Fig. 1L).
We also proved that cells reprogrammed through this strategy maintained genomic integrity even in a compromised disease context, as FA patient cells have a defective DNA repair system (Liu et al., 2014; Raya et al., 2009).
Although ELSO issued guidelines on the use of ECMO in patients with ARDS, these guidelines do not address specific disease context, and are difficult to generalize to the heterogeneous ARDS population.
Whether these activities impart a disease-resolving and/or contributory outcome depends at least in part on the disease context, stage of pathogenesis, and cellular milieu in which these factors are released.
Collectively, these data present a comprehensive profiling of EBV miRNAs in a disease context and implicate their pathological significance.
However the physiological importance of these actin-regulating molecules in DC function in a disease context in vivo has never been reported.
In contrast, the clonal dominance of MDS1 integrated cell populations observed in a recent chronic granulomatous disease (CGD) gene-therapy clinical trials could in part be explained by a lower infused cell dose along with the disease context [4].
Intracerebroventricular delivery of the ASO-tsRNA vector in the SMA mouse model increases SMN protein in the central nervous system of affected animals, demonstrating a platform that can significantly elevate SMN levels in vivo and in a relevant disease context.
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