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Guangping Gao

UMassMed Faculty Page


Title: Screening Novel Clinical Vectors Packaged with Naturally Occurring AAV Capsid Variants from the Human Population with Desired Tissue Tropisms and Properties. UMMS16-71. Patent Pending.

  • The current invention discloses AAV genome sequences that were isolated from normal and diseased human tissue. A novel approach to extract AAV variants allowed for detection of diversity of viral genome. Cap sequences have been determined from the isolates from human biopsies, and include five tissue types and four tumor types. The aggregation of naturally occurring AAV2 and AAV2/3 hybrid variants are unique and have a novel tropism profile suited for tissue targeting purposes.

Title: Use of miR-122 to Treat Liver Diseases. UMMS16-56; Patent Pending.  

  • This invention builds on the new discovery that grainyhead-like 1 and 2 (GRHL-1 and -2) proteins inhibit tumor suppressor, microRNA-122 (miR-122). Increasing the bioavailability of miR-122 may potentially help to treat liver diseases such as steatosis, inflammation, fibrosis, and liver cancer such as hepatocellular carcinoma. Therapeutic inhibition GRHL-1&2 may restore the positive therapeutic effects of miR-122 in liver.

Title: In vitro and in vivo Transduction of Intact Pre-implantation Mammalian Embryos. UMMS 16-29; Patent Pending. 

  • This invention simplifies genetic engineering in mammalian (does it matter which ones?) research and commercial settings by introducing nucleic acids into mammalian embryos without the need for conventional mechanical methods, microinjection or electroporation, which can be lethal. The new transgenic platform works simply by exposing AAV viral particles to an “intact” pre-implantation embryo. Using this method minimizes the need for labor-intensive embryo harvesting and transferring into pseudopregnant females.

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: Gene Therapy and/or Small Molecule Treatment to Correct Beta-Oxidation and Glycolysis in Neurodegenerative Disorders Involving N-Acetylaspartate (NAA) Metabolism and its Associated Metabolic Pathways. UMMS16-17; Patent Pending.

Title: Gene Therapy and/or Small Molecule Treatment to Correct Beta-Oxidation and Glycolysis in Neurodegenerative Disorders Involving N-Acetylaspartate (NAA) Metabolism and its Associated Metabolic Pathways. UMMS16-17; Patent Pending.

Title: Adeno-associated Virus Serotype Vectors Efficiently Transduce Normal Prostate Tissue. UMMS16-16; Patent Pending.

  • Newly discovered rAAVs that can effectively apply gene therapy to the prostate. With direct local injection of rAAV into the prostate, this technology holds the potential to treat numerous diseases, including prostate cancer, without the need for expensive and painful sugary, radiotherapy and medication. 

Title: Repairing Compound Heterozygous Recessive Mutations By Allele Exchange. UMMS16-08; Patent Pending. 

  • This new mutation repair innovation builds on a previously existing two steps gene editing system: 1) induce a break in the genome where the disruption is protective and 2) repair this break with known new mutation. The clever new approach focuses on the first step, which could help patients suffering from genetic diseases caused by two mutations located within a single gene (compound mutation). This concept circumvents the need to utilize the second step of the gene editing technology in these compound mutations because the inventors capitalized on the allele exchange process that occurs in a normal biological setting.

Title: Recombinant Glut1 Adeno-associated Viral Vector Constructs and Related Methods for Restoring Glut1 Expression. UMMS15-61; Patent Pending. 

  • This new rAAV gene therapy introduces Glut1, a primary glucose transporter in the blood brain barrier (BBB), to the brain through endothelial cells lining the brain vasculature. Glut1-deficiency syndrome is an autosomal-dominant disorder, a rare but debilitating childhood neurological disorder. Introducing Glut1 using this technology was shown to improve symptoms associated with Glut1 deficiency in mice, demonstrating potential use in patients with Glut1-deficiency syndrome.

Title: Transgenic Expression of DnaseI in vivo Delivered by an Adeno-associated Virus Vector. UMMS15-38; Patent Pending.

  • This invention uses AAV therapy to deliver an important enzyme, DNaseI, for cyctic fibrosis (CF) treatment. Traditional clinical methods of DnaseI delivery use solely nebulizers, which limit the delivery of the enzyme to the lung. With this new quality-controlled AAV vector for delivery, DnaseI can now be delivered locally to other organ systems affected by CF for more comprehensive disease management.

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. UMMS 15-29; 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: Novel rAAV Genome Designs Using Artificial Hairpin Loop Structures to Replace at least One AAV Inverted Terminal Repeat (ITR). UMMS 15-28; Patent Pending. 

  • This invention capitalizes on the discovery that short hairpin RNA (shRNA) can hinder viral genome replication when specifically placed in relationship to the AAV ITR. To improve the efficacy of AAV-mediated shRNA delivery, the inventors designed and tested multiple rAAV with shRNA placed in different regions of the AAV genome in order to identify the optimal AAV vector for shRNA.

Title: Modified Mullerian Inhibiting Substance (MIS) Proteins and Uses Thereof for the Treatment of Diseases. UMMS14-56; Patent Pending.

  • A new recombinant human MIS protein with improved effectivity may improve the treatment of cancer or neurodegenerative disease. This technology maximizes bioactivity using a leader sequence and a modified cleavage site that promotes increased product yield and MIS processing. This recombinant MIS protein also includes a tag to facilitate purification for research and therapeutic purposes.

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 rAA-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. 

Title: Critical Amino Acid Residues Contribute to Crossing Vascular Barrier by AAV9 Vector. UMMS12-73; UMMS; Patent Pending. 

  • New rAAV vector technology desirable for gene therapy applications addressing muscular and lung disorders. A novel AAV9 capsid mutant produces local tissue-restricted expression and genome persistence, delivered by intravascular (IV), intramuscular (IM) and intranasal (IN) route. The therapy is effective while producing low levels of expression in liver.

Title: SRPX FOR TREATMENT OF CANCER. UMMS 12-08; Patent 9,290,744

  •  This technology provides compositions and methods of treatment for lung cancer. The invention is based on a discovery the tumor suppressor, SRPX is found at low levels of expression in solid human tumors compared to normal tissue. When SRPX is introduced at sufficient levels to tumor cells, SRPX can induce apoptosis and senescence to inhibit cellular proliferation. SRPX can easily be administered using the AAV gene therapy method and markedly suppresses lung cancer in mice.


Title: Chimpanzee-Derived Novel Natural Variants of AAV9: Vector Development and Interrogation of Correlations Between Capsid Structure and Vector Biology. UMMS10-39; Patent Pending.

  • Novel method to isolate and characterize natural variants of AAV9. This new information allows for designing caspids with higher efficiency in packaging and tissue specificity. 


  • New effective and safe gene therapy approach that allows gene delivery across the blood brain barrier (BBB) upon intravascular administration. Designed to minimize off-target effects by incorporation of non-CNS-tissue specific miRNA binding site into the transgene expression cassette.



  • AAV10 vector-based gene therapy for treating ALS with enhanced tropism for neuronal cells. Administration of rAAV encoding inhibitory RNA for superoxide dismutase 1 (SOD1) distributes widely throughout CNS with low toxicity. Long-term inhibition of mutant SOD1 improves lifespan in the animal models.

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.


  • 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 TopicsMultiple SclerosisGene TherapyLiver diseaseFibrosisHepatocellular carcinomaTumor suppressorAAV,  Metabolic inflammationSteatosisResearch ToolsAnimal ModelsGene editingOphthalmologyBlindnessCorneal DiseasesCorneal NeovascularizationNeurologyNeurodegenerative disordersAlzheimer's diseasesCanavan DiseaseMultiple SclerosisCancerProstate cancerCRISPRHeterozygous Recessive MutationCompound mutationGlut1-deficiencyCystic fibrosisDNaseIRNAi/micro-RNA TherapyInfectious DiseaseGene therapy improvementVirologyImmunologyBiologicsLung cancerFamilial hypercholesterolemia (FH)Low density lipoprotein (LDL)DyslipidemiaCardiovascular disease/CardiologyNeuronal SOD1Amyolateral Sclerosis (ALS)