Generation of a WA14 hESC sub-line carrying a hemizygous ABCD1 (C.1696_1710 del) mutation introduced by CRISPR/Cas9 technology.

Wonjun Hong, Wonjin Yun, Wonji Choi, Daryeon Son, Gwonhwa Song, Seungkwon You

Stem cell research 2021 Apr

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ATP-binding cassette transporter subfamily D member 1 (ABCD1) gene is a member of ABC transporter super family, which conduct peroxisomal import of very long chain fatty acid and crucial underlying factor that induces X-linked adrenoleukodystrophy (X-ALD) when the gene is defected. Here, we report the generation of a human embryonic stem cell sub-line harboring a hemizygous ABCD1 mutation (C.1696_1710 del) using CRISPR/Cas9 system. Established line expresses pluripotency marker genes, can be differentiated to three germ layers, and maintains a normal karyotype. Copyright © 2021 The Authors. Published by Elsevier B.V. All rights reserved.

Citation

Wonjun Hong, Wonjin Yun, Wonji Choi, Daryeon Son, Gwonhwa Song, Seungkwon You. Generation of a WA14 hESC sub-line carrying a hemizygous ABCD1 (C.1696_1710 del) mutation introduced by CRISPR/Cas9 technology. Stem cell research. 2021 Apr;52:102244