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Novel epigenome editors for multiplexed gene regulation

Novel epigenome editors for multiplexed gene regulation

Unmet Need Epigenome editors activate or inhibit gene expression without modifying the underlying DNA sequence. Consequently, these editors can reversibly fine-tune gene expression without the risk of permanently altering the genome or off-target editing. Epigenome…

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The Chronic Overlapping Pain Condition Screener

The Chronic Overlapping Pain Condition Screener

Unmet Need Ten Chronic Overlapping Pain Conditions (COPCs) are currently recognized by the National Institutes of Health Pain Consortium (eg, irritable bowel syndrome, chronic migraine headache, and chronic low back pain). These conditions affect millions…

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Novel Self-Assembling Nanoparticles for Extended Duration Local Anesthetics

Novel Self-Assembling Nanoparticles for Extended Duration Local Anesthetics

Unmet Need An estimated 40 million surgeries are performed every year in the United States. Out of this population, an estimated 80% had post-operative pain with 75% reporting it as moderate to extreme. Local anesthetics…

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Olfactory neuroblastoma mouse model

Olfactory neuroblastoma mouse model

Unmet Need Olfactory neuroblasoma (ONB) is an agressive tumor that is thought to arise in basal stem cells in the adult olfactory epithelium. This rare cancer affects 1 in 2.5 million people each year with…

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Methods and compositions for treating protein aggregation diseases

Methods and compositions for treating protein aggregation diseases

Unmet Need Neurodegenerative disease affects more than 50 million people per year. Certain neurodegenerative diseases, like Alzheimer’s disease, result from the accumulation of amyloid-forming proteins in the brain, which results in cognitive impairment and has…

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Mouse model of Alzheimer’s disease

Mouse model of Alzheimer’s disease

Unmet Need Alzheimer’s disease is a neurodegenerative disease marked by loss of cognitive function. There is currently no cure for Alzheimer’s disease. In fact, the limited therapies available only alleviate symptoms for a short time…

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Screening method to identify lead compounds to treat hereditary neurological disorders and other diseases associated with MutSβ activity

Screening method to identify lead compounds to treat hereditary neurological disorders and other diseases associated with MutSβ activity

Unmet Need The human genome is constantly under the threat of DNA damage and replication errors that can result in mutations. A heterodimer of DNA mismatch repair proteins named MutSβ has been linked to a…

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Mouse model of cholinergic dysfunction to evaluate cognitive enhancers for Alzheimer’s disease treatment

Mouse model of cholinergic dysfunction to evaluate cognitive enhancers for Alzheimer’s disease treatment

Value Proposition Defects in acetylcholine transmission have been associated with several major neurodegenerative disorders including Alzheimer’s disease, dementia, cerebral palsy, Parkinson’s disease, Huntington’s disease and myasthenia gravis. Although cholinergic dysfunction has been implicated in these…

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Shank3 knockout mice as a model for autism

Shank3 knockout mice as a model for autism

The Shank3B- knockout allele has a neo cassette replacing the PDZ domain (exons 13-16) of the Shank3 gene, resulting altered expression of the Shank3b isoform. These mice may be useful for studying behavioral abnormalities associated…

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