Title: SerpinA1 Null Mouse as a Model for Alpha-one Antitrypsin Deficiency and Lung Conditions. UMMS15-62. Patent Pending.
The invention provides a novel mouse model with alpha-one antitrypsin deficiency for use as a tool to study lung conditions including emphysema. With the use of the recently developed CRISPR technology, the inventors designed and implemented a strategy to delete all five copies of the SerpinA1 gene at once overcoming previous technical limitations preventing development of SerpinA1 null mice.
Title: MicroRNA Mediated Knockdown of SOD1 Using Recombinant Adeno-associated Vectors. UMMS13-19; Patent Pending.
This invention discloses a novel miRNA delivered by rAAV for silencing SOD1 & C9orf72 SODl genes, which are associated with ALS. This method enables effective therapy at low doses with the persistence of rAAV episomes that continually expresses the nucleic acids, thus rendering re-treatment unnecessary. This method also minimizes rAAV exposure to non-CNS peripheral tissue.
This invention uses a dual-specificity AAV vectors to correct Alpha 1-Antitrypsin (AAT) deficiency. This dual vector carries: 1) miRNAs that target and inhibit the expression of the mutant endogenous protein (AAT), and 2) gene for modified and functional AAT protein that is not targeted by the aforementioned miRNA.
Innovation Topics: Gene Therapy, Research Tools, Animal Models, Pulmonology, CRISPR, Alpha 1-Antitrypsin (AAT) deficiency, Emphysema, Lung disease, RNAi/micro-RNA Therapy, AAV, Amyolateral Sclerosis (ALS), Neuronal SOD1, Gene editing