Nature Communications (Jun 2022)

Establishment of mouse model of inherited PIGO deficiency and therapeutic potential of AAV-based gene therapy

  • Ryoko Kuwayama,
  • Keiichiro Suzuki,
  • Jun Nakamura,
  • Emi Aizawa,
  • Yoshichika Yoshioka,
  • Masahito Ikawa,
  • Shin Nabatame,
  • Ken-ichi Inoue,
  • Yoshiari Shimmyo,
  • Keiichi Ozono,
  • Taroh Kinoshita,
  • Yoshiko Murakami

DOI
https://doi.org/10.1038/s41467-022-30847-x
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 16

Abstract

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Inherited GPI deficiency (IGD) is caused by PIGO mutations. Here, the authors generate a mouse model of IGD and show that AAV-mediate gene therapy, for knock-in as well as extra-chromosomal expression of Pigo cDNA, ameliorates pathology in the mice.