Modulated protein for motor neuron disease treatment
This technology is a strategy for treating spinal muscular atrophy and other motor neuron diseases via modulation of heat shock chaperone protein Hspa8.
Unmet Need: Treatment for motor neuron disease including SMA and ALS
Motor neuron diseases are a devastating group of illnesses for which there is currently no cure. For diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS), the expected survival is typically 2-3 years and there are few effective treatment options. The root causes of both diseases are still unknown, though recent work has shown that RNA-processing proteins may play some role in disease progression. Thus, there remains a great and unmet need for an effective treatment for these motor neuron diseases.
The Technology: Modulated protein for treatment of motor neuron disease
This technology describes the modulation of protein Hspa8, which can be used to treat motor neuron diseases such as spinal muscular atrophy. A single nucleotide change in this specific protein has been shown to be highly protective, shifting its role from chaperone protein towards microautophagy. The modulated protein prevented both motor neuron death and muscle degeneration, ultimately delaying the onset of paralysis and death. This protective effect could potentially be applied across a broad spectrum of motor neuron diseases.
This technology has been validated in a mouse model of spinal muscular atrophy.
Applications:
- Treatment strategy for SMA, ALS, and other motor neuron diseases
- Research tool for studying microautophagy in neurodegenerative disease
- Research tool for studying mechanisms of motor neuron disease initiation and development
- Mouse model for studying protective mutations against neurodegenerative disease
- Neuronal regeneration
Advantages:
- Only single nucleotide change required to gain protective effects
- Prevents motor neuron death and muscle degeneration
- Delays onset and progression of motor neuron disease
Lead Inventor:
Patent Information:
Tech Ventures Reference:
IR CU18201
Licensing Contact: Ron Katz