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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…
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…
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…
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…
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…
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…
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…