4.2 Article

Generation of a homozygous CIITA knockout iPS cell line using the CRISPR-Cas9 system

Journal

STEM CELL RESEARCH
Volume 57, Issue -, Pages -

Publisher

ELSEVIER
DOI: 10.1016/j.scr.2021.102580

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Funding

  1. Fondazione Aiuti per la Ricerca sulle Malattie Rare (ARMR), Bergamo, Italy
  2. Fondazione Telethon - Italy [GGP20073]

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The study successfully generated an isogenic iPSC line with a homozygous frameshift mutation in the CIITA gene using CRISPR-Cas9 technology. The CIITA(-/-) iPSCs exhibited typical pluripotent cell characteristics, normal karyotype, and differentiation potential.
Human induced pluripotent stem cells (iPSCs) have great promise in regenerative medicine. However, several limitations, including immune-incompatibility, have raised concerns regarding their clinical application. Recent studies have shown that human iPSCs and their derivatives lose their immunogenicity when major histocompatibility complex (MHC) class I and II genes are inactivated and CD47 is over-expressed. In this study, we used CRISPR-Cas9 technology to generate an isogenic iPSC line with a homozygous frameshift mutation in the MHC II transactivator (CIITA) gene. The CIITA(-/-) iPSCs exhibit typical morphology of pluripotent cells, normal karyotype, expression of pluripotency markers and differentiation capacity in the three germ layers.

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