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The fact that the syndrome presents a range of severity and variability in its clinical manifestations and a high genetic heterogeneity makes the development of disease models difficult, which have been limited to specific mutations

Addressing any fungal or bacterial overgrowth will also ensure the tight junctions stay repaired

doi: 10.1016/j.cmet.2013.11.005

doi: 10.1093/ijnp/pyz052 [DOI] [PMC free article] [PubMed] [Google Scholar] 169

AON design for NF1 exon skipping and evaluation of exon skipping efficacy For AON design, exon 2 of NF1 was selected as the target, and 13 different AON sequences were designed using Human Splicing Finder and Examine the Secondary Structure of mRNA software

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