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Terence R Flotte





UMassMed Faculty Page



  • 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.

Title: AAV’S AND USES THEREOF. UMMS09-58; Patent  8,734,8099,284,357 

  • Novel AAVs with tissue targeting capability for application in therapeutic gene therapy and research purposes. With efficient delivery, these AAVs have minimal toxic effects on local tissues. They can also be used to develop somatic transgenic animals with a tissue specific promoter. Additionally, this invention includes composition and a method for isolating other novel AAVs. This technology can be used to express RNAi or RNAi sponge that inhibits one or more RNAi functions in a tissue.

Title: Isolation of Novel AAV Sequences from Tissue RNAs by Reverse Transcription (RT)-PCR. UMMS08-56; Patent Pending.  

  • This new innovation consists of improvements to the current gene therapy methods using adeno-associated virus. By confronting the issues associated with tissue specificity, the inventors have isolated novel capsid sequences from nonhuman primate tissue that harbor low levels of AAV. The invention discloses a novel method for isolating tissue specific AAV capsid sequences for high efficacy and tissue specificity of AAV that may be valuable in multiple contexts.


Innovation TopicsGene TherapyAlpha 1-Antitrypsin (AAT) deficiencyAAVGene editingAnimal ModelsGene therapy improvement