Christian Mueller

 

 

 

 

UMassMed Faculty Page

Inventions:  

Title: SerpinA1 Null Mouse as a Model for Alpha-one Antitrypsin Deficiency and Lung Conditions. UMMS15-62. Patent Pending. 

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. 

Title: RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES. UMMS11-45; Patent 9,226,9769,885,057.

  • 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 TopicsGene TherapyResearch ToolsAnimal ModelsPulmonologyCRISPRAlpha 1-Antitrypsin (AAT) deficiency EmphysemaLung diseaseRNAi/micro-RNA TherapyAAVAmyolateral Sclerosis (ALS)Neuronal SOD1Gene editing