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One or more keywords matched the following properties of Vandenberghe, Luk
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overview The Vandenberghe laboratory deconstructs viral evolution to improve viral vector design and use in gene therapy. We aim to more profoundly understand capsid structure-function relationships and use this information to select and engineer gene therapy vectors for clinical indications. For many of these studies, we use the ssDNA mammalian adeno-associated virus (AAV) as a model. AAV is not only one of the smallest and simplest mammalian viruses, it has also captivated the world of gene therapy as AAV, with remarkable efficiency and safety, is able to deliver and transduce genetic cargo into therapeutic target tissues such as the retina, cochlea, liver, and CNS. AAVs come in many flavors that were originally distinguished based on serology, but now are more and more phylogenetically and structurally defined. Different AAVs demonstrate very distinct phenotypes (tropism, receptor use, host response, assembly, etc.) as a wild type virus or replication-defective vector. We are interested in functionally and structurally understanding the mechanism of these stark differences from the perspective of virus as well as host. One avenue towards this goal is to study the evolutionary biology of this virus, and use mathematical, statistical, and systems approaches in combination with empirical and molecular methods to shed light on the structure-function relationship of these small, yet intricately complex infectious protein assemblies. We aim at integrating these findings into improved technologies for therapeutic gene transfer, and use these findings and novel reagents toward building innovative therapies for diseases with unmet need, with a particular focus on blinding and hearing disorders. lab webpage: http://www.vdb-lab.org/ Gene Transfer Vector Core: http://vector.meei.harvard.edu/
One or more keywords matched the following items that are connected to Vandenberghe, Luk
Item TypeName
Academic Article Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis.
Academic Article Gene therapy for deaf mice goes viral.
Academic Article Tailoring the AAV vector capsid for gene therapy.
Academic Article Novel adeno-associated viral vectors for retinal gene therapy.
Academic Article Retinal gene therapy coming of age.
Academic Article Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey.
Academic Article Progress in gene therapy for neurological disorders.
Academic Article Progress in gene therapy for neurological disorders.
Academic Article Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
Academic Article Perspectives on best practices for gene therapy programs.
Academic Article What Is Next for Retinal Gene Therapy?
Academic Article In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector.
Academic Article Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c.
Academic Article Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.
Academic Article Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction.
Academic Article Breaking and Sealing Barriers in Retinal Gene Therapy.
Academic Article Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration.
Academic Article MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
Search Criteria
  • Gene Therapy
Funded by the NIH National Center for Advancing Translational Sciences through its Clinical and Translational Science Awards Program, grant number UL1TR002541.