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Using Hetero-multivalent DNA-functionalized Materials to Detect Genetic Mutations
Application Improving diagnostic accuracy using hetero-multivalent DNA-functionalized materials for disease targets containing one or more genetic mutations. Key Benefits Tunable oligos increase specificity and cooperative binding to the disease target of interest. Distinguishes between cis- and trans mutations. Identifies different strains of...
Published: 3/1/2024       Contributor(s): Brendan Deal, Khalid Salaita, Rong Ma, James Kindt
Novel Therapeutic Target for Fragile X Syndrome and Neurodevelopmental Disorders
Application Salvianolic acid C for treating Fragile X Syndrome (FXS). Key Benefits New therapeutic drug candidate for the treatment of FXS. Preliminary data in the laboratory in organoids treated with the drug showed rescued cell phenotypes known to be associated with the disease in humans. Potential to treat other neurodevelopmental disorders. Market...
Published: 3/1/2024       Contributor(s): Peng Jin, Chuan He, Zhongyu Zou
Whole-Animal Genetic Model to Mimic Aspects of the Patient Phenotype of Classic Galactosemia
­ Application An animal model for studying the underlying causes of galactosemia and screening for novel drugs to treat the condition. Key Benefits The first animal model for elucidating the underlying causes of galactosemia in infants. Animals with galactosemia causing genetic knockout exhibit similar disease phenotypes and complications...
Published: 7/28/2023       Contributor(s): Judith Fridovich-Keil
Cas9 Controlled Gene Activation
Application Flexible system using Cas9 to modulate gene expression. Key Benefits Edits genes by cleaving DNA as well as inhibiting transcription. No need to transfect cells with additional Cas9. Market Summary The CRISPR-Cas9 system is a bacterial adaptive immune system that has been appropriated for genetic engineering. The CRISPR-Cas9 system...
Published: 12/1/2023       Contributor(s): David Weiss, Hannah Ratner
3q29 Deletion Mice
Application Mouse models with the deletion of 3q29 for the study of neurological disorders such as schizophrenia, autism, and anxiety disorders. Technical Summary Copy Number Variants (CNV’s) are large genomic deletion or duplications. One of the CNV’s, the 3q29 deletion, confers more than 40 fold increase in risk of schizophrenia. Most...
Published: 7/28/2023       Contributor(s): Tamara Caspary, David Weinshenker, Stephen Theodore Warren
Human Induced Pluripotent Stem Cells
ApplicationHuman induced pluripotent stem cells useful for human stem cell biology, Lesch-Nyhan disease or DYT1 dystonia.Technical SummaryLesch-Nyhan syndrome (juvenile gout) is a rare, inherited disorder caused by deficiency in hypoxanthine-guanine phosphoribosyltransferase (HGPRT). The disease causes overproduction of uric acid often leading to gout...
Published: 7/28/2023       Contributor(s): Hyder Jinnah
Combination Drug Treatment for Neoplasms Associated with Tuberous Sclerosis
Application Combination of rapamycin and imatinib for the treatment of neoplasms associated with tuberous sclerosis (TS). Key Benefits Combined therapy shows 97% decrease in tumor volume in mouse model of TS compared to vehicle treatment. Dual therapy blocks two major signaling pathways implicated in TS. Market Summary Tuberous sclerosis (TS)...
Published: 9/18/2023       Contributor(s): Jack Arbiser
Compounds Show Promise in Suppressing Fragile X Toxicity
Application Compounds for the treatment of Fragile X-associated tremor/ataxia syndrome. Key Benefits 11 known, FDA approved drugs identified that reduced rCGG-mediated lethality to a certain degree. 3 of these drugs also rescued locomotion deficits. Technical Summary Fragile X syndrome (FXS) is the most common inheritable form of cognitive...
Published: 9/18/2023       Contributor(s): Peng Jin, Abrar Qurashi