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Miguel S Sena Esteves





UMassMed Faculty Page


Title: Method to Enhance the Efficiency of Systemic AAV Gene Delivery to the Central Nervous System. UMMS16-19; Patent Pending.

  • This invention relates to compositions and methods for increased efficiency of gene transfer to the CNS. The invention is based on the surprising discovery that viral vector-mediated delivery of nucleic acids to the CNS of a subject can be enhanced by administering the viral vector (rAVV) with a composition comprising a highly hydrophilic molecule conjugated to a blood brain barrier (BBB)-receptor ligand (e.g., K16ApoE). 

Title: New AAV Vectors for Safe and Efficient Expression of Lysosomal Enzymes in the CNS. UMMS15-19; Patent Pending.

  • New AAV vectors that enable transgene expression at therapeutic levels for treatment of lysosomal and other disorders. These levels are achieved without the adverse events that arises due to secondary effects of AAV- mediated product delivery. This newly engineered regulatory element can apply broadly to circumvent adverse effects of AAV therapy.  

Title: Novel High Efficiency Library-identified CNS-tropic AAV Vectors 2. UMMS14-59. Patent Pending.

  • Newly designed recombinant AAV capsids with greater efficiency for CNS delivery than AAV9, the natural variant with the currently highest known CNS transduction efficiency. These new AAV capsids, B1-B4, show considerably lower off-target effects in the liver when compared to AAV9. Additionally, each of these AAV capsids show selectively higher efficiency for specific peripheral tissues (e.g. pancreas, sk. muscle, heart, adipocyte). 

Title: Novel High Efficiency Peptide Grafted CNS-tropic AAV Vectors 1. UMMS14-58. Patent Pending.

  • New AAV technology with an insertion of 19-amino acid “alanine string” that has dramatically increased CNS tropism compared to that of AAV9, the natural variant with the currently highest known CNS transduction efficiency. This technology shows increased CNS transduction without changing transduction efficiency of peripheral tissues. 


Innovation Topics: Gene TherapyAAVNeurodegenerative disordersNeurologyGene therapy improvementNeurological lysosomal disordersMetaboendocrineObesityGene editing