TS or not TS?

Athappily, Cyriac; Patani, Rickie; Chawda, Sanjiv; Rosser, Elisabeth; Silva, Rajith de

doi:10.1136/practneurol-2012-000371

Cited by 1 publication

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“…These approaches and their practical utility in patient-specific hiPSCs have been recently reviewed elsewhere (Hendriks et al 2016 ). Genome editing technologies are particularly relevant to monogenic diseases rather than complex genetic disorders or sporadic disease (Shribman et al 2013 ; Samani et al 2015 ; Patani et al 2013 ; Athappily et al 2013 ). An ideal approach for mendelian disorders is to generate reciprocal isogenic lines for a given mutation being studied—i.e., the mutation is corrected to create one isogenic pair, and it is separately inserted into a control line to generate a second isogenic pair (Liu et al 2012 ).…”

Section: Genomic Variation Between Hipsc Linesmentioning

confidence: 99%

The translational potential of human induced pluripotent stem cells for clinical neurology

Devine

Patani²

2016

Cell Biol Toxicol

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The induced pluripotent state represents a decade-old Nobel prize-winning discovery. Human-induced pluripotent stem cells (hiPSCs) are generated by the nuclear reprogramming of any somatic cell using a variety of established but evolving methods. This approach offers medical science unparalleled experimental opportunity to model an individual patient’s disease “in a dish.” HiPSCs permit developmentally rationalized directed differentiation into any cell type, which express donor cell mutation(s) at pathophysiological levels and thus hold considerable potential for disease modeling, drug discovery, and potentially cell-based therapies. This review will focus on the translational potential of hiPSCs in clinical neurology and the importance of integrating this approach with complementary model systems to increase the translational yield of preclinical testing for the benefit of patients. This strategy is particularly important given the expected increase in prevalence of neurodegenerative disease, which poses a major burden to global health over the coming decades.

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