Science and Research

Generation of two TMEM16A knockout iPSC clones each from a healthy human iPSC line, from a Cystic Fibrosis patient specific line with p.Phe508del mutation and from the gene corrected iPSC line

The Transmembrane member 16A (TMEM16A), also known as anoctamin-1 (ANO1), is a calcium-activated chloride channel present in the airway epithelium. It is known to be involved in the apical chloride secretion indicating that TMEM16A could be addressed for the treatment of chloride secretion defects like in Cystic- Fibrosis (CF). In this paper we generated knockout cell lines using CRISPR/Cas9-mediated ablation in a healthy human iPSC line (MHHi001-A), in a CF patient iPSC line (MHHi002-A) and in its corrected counterpart (MHHi002-A-1). These lines can be used for gaining information about the role of TMEM16A for mucus secretion and/or production and evaluating its therapeutic potential.

  • Jaboreck, M. C.
  • Lühmann, J. L.
  • Mielenz, M.
  • Stanke, F.
  • Göhring, G.
  • Martin, U.
  • Olmer, R.
  • Merkert, S.
Publication details
DOI: 10.1016/j.scr.2022.102918
Journal: Stem Cell Res
Pages: 102918 
Work Type: Original
Location: BREATH
Disease Area: CFBE
Partner / Member: MHH
Access-Number: 36162332

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