VIRAL RNA FOR TREATING NEURODEGENERATION

Professor John Sinclair and Dr Matthew Reeves have found that a non-coding viral
RNA has the potential to:
• Treat a range of neurodegenerative diseases by preventing neuronal death
• Directly target neuronal cells undergoing apoptosis or at risk of cell death
• Cross the blood-brain barrier when complexed with an appropriate targeting
moiety

Potential Uses

• Disease modifying therapy for neurodegenerative disorders including Parkinson’s,

Alzheimer’s and Huntington’s disease and Amyotrophic Lateral Sclerosis
• Protects neuronal cells from cell death in cell-based models of neurodegenerative
disorders
• Neuroprotection in a range of model systems of dopaminergic cell loss mimicking
Parkinson’s disease

For further information please contact:

Dr Rachel Atfield
rachel.atfield@enterprise.cam.ac.uk
 +44 (0)1223 760339
Cambridge Enterprise Limited, University of Cambridge
The Hauser Forum, 3 Charles Babbage Road, Cambridge CB3 0GT UK
www.enterprise.cam.ac.uk

Case Ref: Sin-1808-06

Background
Commercialisation and patent status
Neurodegenerative disease results from the
We are seeking commercial partners for licensing,
deterioration of neurons, which over time leads to
collaboration and development of this technology.
dysfunction and disabilities. Parkinson’s disease
This technology is protected by PCT application
is a common neurodegenerative disorder of the
WO2009/141625.
CNS in which there is selective loss of neuronal
populations. The disease is also often associated
with mitochondrial dysfunction and oxidative
stress. Many lines of evidence converge on a final Figure 1: Tail vein delivery of Peptide/beta 2.7
common pathway in sporadic and familial forms of RNA complex ameliorates the behavioural deficits
Parkinson’s Disease that involve a mitochondrial in a Parkinson’s rat model compared to animals
component and therapeutic targeting of this receiving Peptide/actin treatment by a)
element may have major implications in amphetamine-induced rotation (test of lesion
preventing or slowing down this disorder. severity) b) apomorphine-induced rotation (test of
striatal dopaminergic function) c) cylinder test
(examination of spontaneous use of contralateral
Technology
forelimb) and d) stepping test (test of
Professor John Sinclair and Dr Matthew Reeves sensorimotor function).
in the Department of Medicine at the University of
Cambridge have identified a novel 2.7 kilobase
virally-encoded RNA expressed during human
cytomegalovirus infection which does not encode
for any known protein and functions to prevent
cell death (Science 2007). This RNA has been
shown to rescue neuronal cells from death in
rotenone and 6-OHDA induced cell-based models
of neurodegenerative disorders.

In collaboration with scientists at the Brain Repair

Centre in Cambridge the team has shown that
direct injection of the protective RNA into the
substantia nigra of a rat model of Parkinson’s is
protective. They have also shown that tail vein
delivery of the RNA with a targeting moiety that is
able to cross the blood brain barrier efficiently
protects lesion-induced behavioural deficits in a
rat model of Parkinson’s (Figure 1) and that this
RNA is clearly present in rat brain sections up to
10 days after tail vein delivery.

Publication
Complex I binding by a virally encoded RNA
regulates mitochondria-induced cell death.
Reeves et al. Science (2007). 316(5829):1345

Sin-1808-06