Protection of the mouse brain by microRNA-7 against the toxicity of a-synuclein (A) Tyrosine hydroxylase (TH) stained coronal section of mouse brain injected in the right striatum with AAV-miR-7 or non-targeting (NT) vector following by a-synuclein preformed fibrils (PFF) or PBS. (B) Substantia nigra sections. (C) Nigral dopamine neuron count. (D) Striatal TH density. (E) Performance on the rotarod.
Parkinson’s disease (PD) is a common neurogenerative disorder that affects approximately 1% of the population over the age of 60. The characteristic disabling motor abnormalities in PD are due to progressive loss of dopaminergic neurons. Abnormal accumulation of misfolded α-Synuclein is associated with neuronal death that leads to PD. Regulating α-Synuclein expression through microRNA (miRNA) is a promising therapeutic strategy for disease modification in PD and related Synucleinopathies.
Rutgers scientists have identified several miRNAs with potential for treating neurodegenerative diseases such as PD. These include miRNA-7, miRNA-153, miRNA-34b, etc. For example, miRNA-7 is enriched in the brain and binds to the 3’-UTR of α-Synuclein mRNA in a sequence specific manner and inhibits its translation. The role of miRNA-7 as a regulator of α-Synuclein expression and its neuroprotective effects have been demonstrated in a mouse model of α-Synucleinopathy. Hence, down-regulation of α-Synuclein expression by miRNA-7 and other related miRNAs could be exploited in a gene therapy-based approach for the treatment of PD.
- Highly specific in targeting α-Synuclein
- Potential for better safety profile due to endogenous nature of miRNA’s
- High tissue-specificity
- Therapeutics for Parkinson’s disease
- Treatment for other α-Synucleinopathies, such as dementia with Lewy bodies and multiple system atrophy
Intellectual Property & Development Status:
- Issued patents: US10,184,123, US9,708,611, US9,255,266
- Available for licensing and/or research collaboration.