About the invention

The mitochondrial translocator protein (18 kDa) (TSPO) knockout (C57BL/6-Tspotm1GuWu) is a mouse model developed at ANSTO, in collaboration with The University of Sydney. 
 

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Its purpose is to enable the conclusive testing of selectivity and mode of action of TSPO-binding compounds with diagnostic and therapeutic potential. This approach can be applied to the development of new chemical entities as well as the repurposing of existing drugs.
 

For the first time, observations made in reductionist models, such as cell culture, can be translated to a widely used animal model of human physiology and verified unambiguously in a complex mammalian system.
 

The paradigm shift

 

Pathways of the innate immune system are involved across many acute and chronic diseases
 

Understanding inflammatory or immune-mediated pathways allows mechanism-based diagnosis and therapy across organ/systems and disease classifications
 

Broader indications for drugs targeting these pathways increases clinical return on investment

 
This TSPO knockout mouse model closes an important gap in the tool kit available to study conditions in which the TSPO or TSPO-binding compounds are physiologically relevant and promise therapeutic potential, including:
 

  • Behavioural and psychiatric disorders, such as anxiety disorders
  • Obesity, diabetes and metabolism disorders
  • Neurodegenerative disorders, such as Alzheimer’s disease and Parkinson’s disease
  • Inflammatory disease, such as multiple sclerosis (MS)
  • Inflammation-associated pain
  • Cancer biology

 

“The paper of R.B. Banati and co-workers is definitely a breakthrough… (it) rejuvenates basic science around TSPO and the potential design and development of drugs interacting with this target.”

 

Gerard Le Fur
Academie des Sciences, France