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Phillip D Zamore






UMassMed Faculty Page


Title: Development of Anti-angiogenic miRNA Therapeutics for Corneal Neovascularization. UMMS 16-23; Patent Pending. 

  • This new technology includes two novel inventions: 1) discovery of miRNAs that influences the neovascularization of the cornea and 2) optimization of rAAV gene therapy method for specific delivery to the cornea. Neovascularization is the most common corneal pathological condition and underestimated cause of blindness. Injection of the rAAV constructs that include synthetic nucleic acids that mimic or inhibit the discovered miRNA can reduce corneal neovascularization in mice. 

Title: Nucleic Acid-guided, Thermostable Restriction Enzyme for DNA and RNA. UMMS15-41.

  • Our invention is a method for the creation of a thermostable, programmable restriction enzyme that can be used to cleave ssRNA, ssDNA, dsRNA, or dsDNA at a specific site determined by the sequence of the small DNA guide bound to Thermus thermophilus Argonaute (TtAgo) protein. This invention is a method that can be used as a molecular biology research tool for cloning and/ manipulating DNA or RNA for research use. 

Title: Transient Delivery of Designer Nuclease Protein Using Viral Vectors For Safer Gene Editing. UMMS15-34; Patent Pending. 

  • This new technology addresses the fundamental issues arising in the emerging biotechnology of gene editing, off-target effects from prolonged treatment. Specifically, this invention improves the CRISPR gene editing system by delivering its nuclease component Cas9 by using a traditional gene therapy method utilizing AAV. Once nuclease Cas9 is delivered, the Cas9 will slowly degrade and yield a reduction in genotoxicity and undesirable off-target genome editing associated with CRISPR therapy. 

Title: Development of Efficient and Safe rAAV Compatible Silencing Construct. UMMS15-29; Patent Pending.

  • This invention spans from the invention of UMMS15-28, an rAAV optimized for compatibility and efficacy for shRNA delivery. This process was tested in UMMS15-28 by examining the regional importance of shRNA within the viral genome. This upgraded shRNA-rAAV delivery system has optimized flanking sequences and shRNA structure. The shRNA backbone has lower loop complementarity, causing lower thermodynamic stability that increases its efficacy. This concept was tested by designing multiple Artificial miRNA (AmiRNA), where AmiRNA have the optimized structure while still possessing the shRNA interfering properties. This mechanism for shRNA deliver is highly efficient and safe for sustained silencing. 

Title: Position specific requirements for nucleotide pairing or mismatches between microRNA or small interfering RNA (siRNA) and target RNA to promote occupancy and retention of an Argonaute protein or RNA-induced Silencing Complex (RISC) on a target RNA. UMMS14-70; Patent Pending.

  • This new invention of “tether” oligo allows for siRNA delivery with higher potency and tissue specificity in medical applications. Tethering endogenous miRNAs that are highly tissue specific can direct them to the target mRNA of interest and this nuclease-resistant oligo can robustly increase efficacy of siRNA therapy. Unlike previous methods, this oligo restricts siRNA to specific cell types which may yield reduced off-target effects. 

Title: miRNA-mediated Transcriptional Detargeting to Reduce Transgene Immunity. UMMS14-22; Patent Pending.

  • This new AAV technology involves co-delivery of a transgene that minimizes immune responses against the transgene product of interest. Specifically, this process involves administering a rAAV-harboring a transgene engineered to express an inhibitory RNA transcript that targets one or more immune- associated miRNA. This interaction lowers the immune response and consequently increases the potency of the therapeutic protein and its effects. 


  • 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. 



  • This invention discloses a method for studying mRNA sequences without disrupting transcript connectivity or relative abundance. Contrary to existing methods to study mRNA splicing, this method directly assesses the relative abundance of isoforms, with information regarding multiple ligases and mRNA exon connectivity. Improved understanding of isoforms may facilitate further study of developmental disease or cancer.


  • The technology utilizes methods of implementing viral vectors harboring a transgene(s) in combination with tissue specific anti-miRNA sequences to minimize off-target effects. In addition, this method allows for production of somatic transgenic animal models by targeted destruction of specific cell types.


Innovation TopicsGene TherapyAAVOphthalmologyBlindnessCorneal DiseasesCorneal NeovascularizationResearch ToolsRestriction enzymeCRISPRGene editingRNAi/micro-RNA TherapyInfectious DiseaseAAVGene therapy improvementImmunologyAlpha 1-Antitrypsin (AAT) deficiencyFamilial hypercholesterolemia (FH)Low density lipoprotein (LDL)DyslipidemiaCardiovascular disease/Cardiology, Genomics , Transcriptomics, Gene expressionAnimal Models