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Molecular by-pass therapy for mitochondrial dysfunction - Proof of Concept


Defects in the primary metabolic functions of mitochondria, the cell’s
‘power-plants’, underlie a diverse range of human pathologies, including
rare neuromuscular syndromes, many cases of common multifactorial
diseases (e.g epilepsy, deafness, diabetes), neurodegenerative
conditions such as Parkinson’s disease (PD), and devastating metabolic
disorders of infancy. Mitochondrial dysfunction is also a major cause of
tissue damage in heart attack and stroke, and is involved in
cancer progression. This project will facilitate an R&D effort to
develop a common genetic therapy for this vast range of diseases, taking
advantage of the fact that lower organisms possess a built-in
biochemical mechanism for relieving the stress caused by mitochondrial
dysfunction, using alternative respiratory chain enzymes that simply
by-pass the problem. Under ERC Project 232738 (MITO BY-PASS) we have
succeeded in expressing the alternative oxidase AOX from the sea
squirt Ciona intestinalis in mammalian cells and transgenic flies. In
the fruit-fly, AOX expression overcomes the lethality of poisons
directed at mitochondria, and cures flies of genetic defects that
produce features equivalent to PD or fatal mitochondrial diseases of
infancy. Preliminary data on mammalian models also indicates that the
gene can be safely expressed, and contributes similar benefits. These
now constitute a key validation tool to test the potential for AOX
therapy of many human diseases. This PoC project will undertake the
crucial next steps in the development of AOX therapy by implementing a
partner search and negotiating terms for testing the technology in many
different mammalian models of common diseases associated with
mitochondrial dysfunction, and by soliciting major investment for
the development of AOX therapy from both public and private-sector partners.

Call for proposal

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Kalevantie 4
33014 Tampere

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Activity type
Higher or Secondary Education Establishments
Administrative Contact
Susanna Airila (Ms.)
EU contribution
No data

Beneficiaries (1)