Publication:
Generation of transgene-free iPSC lines from three patients with Friedreich's ataxia (FRDA) carrying GAA triplet expansions in the first intron of FXN gene

Thumbnail Image

School / College / Institute

Organizational Unit
GRADUATE SCHOOL OF HEALTH SCIENCES
Upper Org Unit
Organizational Unit
Organizational Unit
SCHOOL OF MEDICINE
Upper Org Unit

Program

KU Authors

Co-Authors

Publication Date

Language

Embargo Status

NO

Journal Title

Journal ISSN

Volume Title

Alternative Title

Abstract

Friedreich's ataxia (FRDA) is a rare neurodegenerative disorder which is caused by triplet repeat expansion (GAA) in the first intron of FXN gene. In this present study, we generated induced pluripotent stem cells (iPSC) lines from fibroblasts of three unrelated FRDA patients using integration-free episomal vectors. All iPSC lines express the pluripotency markers such as OCT4 and SSEA4, display normal karyotypes and can differentiate into all three germ layers via in vivo teratoma formation assay.

Source

Publisher

Elsevier

Subject

Cell biology, Biotechnology, Applied microbiology

Citation

Has Part

Source

Stem Cell Research

Book Series Title

Edition

DOI

10.1016/j.scr.2021.102438

item.page.datauri

Link

Rights

Copyrights Note

Endorsement

Review

Supplemented By

Referenced By

2

Views

4

Downloads

View PlumX Details