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Generation of an isogenic, gene-corrected iPSC line from a pre-symptomatic 28-year-old woman with an R406W mutation in the microtubule associated protein tau (MAPT) gene.

Abstract：

:Frontotemporal dementia with parkinsonism linked to chromosome 17q21.2 (FTDP-17) is an autosomal-dominant neurodegenerative disorder. Mutations in the MAPT (microtubule-associated protein tau) gene can cause FTDP-17, but the underlying pathomechanisms of the disease are still unknown. Induced pluripotent stem cells (iPSCs) hold great promise to model FTDP-17 as such cells can be differentiated in vitro to the required cell type. Furthermore, gene-editing approaches allow generating isogenic gene-corrected controls that can be used as a very specific control. Here, we report the generation of genetically corrected iPSCs from a pre-symptomatic carrier of the R406W mutation in the MAPT-gene.

journal_name

Stem Cell Res

journal_title

Stem cell research

authors

Nimsanor N,Poulsen U,Rasmussen MA,Clausen C,Mau-Holzmann UA,Nielsen JE,Nielsen TT,Hyttel P,Holst B,Schmid B

doi

10.1016/j.scr.2016.09.024

subject

Has Abstract

pub_date

2016-11-01 00:00:00

pages

600-602

issue

3

eissn

1873-5061

issn

1876-7753

pii

S1873-5061(16)30136-2

journal_volume

17

pub_type

杂志文章

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