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Human induced pluripotent stem cell (hiPSC) technology has widely been used for modeling of various genetic diseases. In the disease-modeling iPSC studies, it is necessary to generate hiPSC lines from peripheral tissues of multiple patients and of multiple age- and sex-matched control individuals in order to minimize the differences in genetic background that can affect the results. However, we can generate disease model-hiPSC line with mutant allele being expressing and normal control-hiPSC line with normal allele being expressing from a patient with an X-linked dominant disease such as Rett syndrome, taking advantage of use of the genetic nature (X-chromosome inactivation) and the hiPSC nature (monoclonal proliferation). Furthermore, it may be a straightforward way to identify drug candidates that normalize or minimize the abnormal features and gene expression patterns by a comparative study between mutant active- and normal active-hiPSC lines. Such drug-screening strategy can also be applicable to more major X-linked recessive disease such as Duchenne muscular dystrophy and Fabry disease by generating hiPSC lines from fibroblasts from the heterozygous females. Therefore, X-linked diseases may be efficient targets for drug screening using hiPSC derived from the patients, since faster drug screening hiPSC studies can be conducted for X-linked diseases compared with autosomal diseases by utilizing the genetic natures and the hiPSC nature.

Advantage in Human Induced Pluripotent Stem Cells Research of X-Linked Genetic Diseases for Drug Screening