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GLI-Similar 3(GLIS3) Knock Out (KO) Mice as Models to Screen Therapeutics for Diabetes, Polycystic Kidney Disease, and Hypothyro

Posted May 15 2012 8:00pm

Description of Invention:
GLI-similar (Glis) 1-3 proteins constitute a subfamily of the Krüppel-like zinc finger transcription factors that are closely related to the Gli family. Mutations in human GLIS3 have been implicated in a syndrome characterized by neonatal diabetes and congenital hypothyroidism (NDH) and in some patients accompanied by polycystic kidney disease, glaucoma, and liver fibrosis. To further identify and study the physiological functions of GLIS3, NIEHS investigators generated mice in which GLIS3 is ubiquitously knocked out (GLIS3-KO) or conditionally knocked out in a cell type-specific manner. GLIS3-KO mice develop polycystic kidney disease, hypothyroidism, and neonatal diabetes, as indicated by the development of hyperglycemia and hypoinsulinemia. The pancreatic endocrine cells, particularly insulin-producing pancreatic beta cells, are greatly diminished in these mice. The pancreas-selective knockout mice GLIS3(Pdx1-Cre) develop severe diabetes within 2-3 months, much later than the GLIS3-KO mice. The kidney-selective knockout of GLIS3 (GLIS3(Ksp-Cre) mice lack expression of GLIS3 in the collecting ducts and develop severe polycystic kidney disease within a period of 2-4 months. These mice can be used as models to screen therapeutics for diabetes, polycystic kidney disease, and hypothyroidism.

  • Therapeutic target in the management of diabetes, polycystic kidney disease, and hypothyroidism
  • Models to test therapeutic drugs for diabetes, polycystic kidney disease, and hypothyroidism

  • Provides opportunity to discover upstream signals that regulate GLIS3 activity
  • Can be used in stem cell therapy in diabetes treatment
  • Excellent model to study the role of GLIS3 in neonatal diabetes

Development Status:
  • Early-stage
  • Pre-clinical
  • In vivo data available (animal)

Anton M Jetten (NIEHS)
Hong Soon Kang (NIEHS)
Kristin N Lichti-Kaiser (NIEHS)

Patent Status:
HHS, Reference No. E-303-2011/0

Research Tool — Patent protection is not being pursued for this technology.

Related Technologies:

HHS Reference No. E-253-2010/0 — An In-Vitro Cell System Useful for Identification of RORgamma Antagonists
HHS Reference No. E-222-2009/0 — RORgamma (RORC) Deficient Mice Which Are Useful for the Study of Lymph Node Organogenesis and Immune Responses

Relevant Publication:
  1. Kang HS, et al. [ PMID 19805515 ]
  2. Kang HS, et al. [ PMID 19273592 ]

Collaborative Research Opportunity:
The NIEHS is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize GLIS3 Knock Out Mice. For collaboration opportunities, please contact Elizabeth M. Denholm, Ph.D. at .

For Licensing Information Please Contact:
Suryanarayana Vepa Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325,
Rockville, MD 20852
United States
Phone: 301-435-5020
Fax: 301-402-0220

Ref No: 2439

Updated: 05/2012

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