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Gao, Guangping
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overview

Academic Background

B.A. West China Univ. of Med. Sciences, Chengdu, China
M.S. Florida International University, Miami, FL
Ph.D. Florida International University, Miami, FL
Postdoctoral training at the University of Pennsylvania - School of Medicine

Overview:

Research in the Gao Lab primarily involves the discovery, development and use of adeno-associated virus vectors for gene therapy of genetic diseases and the study of miRNA functions in mammals. The lab works on isolation, characterization and vectorology of novel AAV vectors from primate tissues, molecular mechanisms of AAV evolution and diversity, and molecular interactions between endogenous AAV, AAV vector, host genomes and innate RNAi defense pathways. We also develop novel strategies for rAAV gene therapy of an inherited neurodegenerative disease, Canavan Disease, using novel AAVs that can cross the blood-brain-barrier for efficient CNS gene delivery and endogenous miRNA-mediated posttranscriptional de-targeting. Another area of research interest in the lab is to explore AAV vectors for delivery of denovo synthesized biological miRNA antagonists or over expression of microRNAs to elucidate micro RNA functions in adult mammals.
Summary Focus: Adeno-associated virus vector mediated gene transfer for gene therapy, gene therapy of inherited neurodegenerative Canavan Disease
One or more keywords matched the following items that are connected to Gao, Guangping
Item TypeName
Academic Article Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production.
Academic Article Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system.
Academic Article Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction.
Academic Article Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer.
Academic Article Retinal gene delivery by rAAV and DNA electroporation.
Academic Article Gene therapy for Canavan's disease takes a step forward.
Academic Article Gene transfer to the CNS using recombinant adeno-associated virus.
Academic Article Gene transfer in the liver using recombinant adeno-associated virus.
Concept Genetic Therapy
Academic Article A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
Academic Article The potential of adeno-associated viral vectors for gene delivery to muscle tissue.
Academic Article State-of-the-art human gene therapy: part I. Gene delivery technologies.
Academic Article State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.
Academic Article Adeno-associated virus-mediated microRNA delivery and therapeutics.
Academic Article Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.
Academic Article Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.
Academic Article Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.
Academic Article Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.
Academic Article Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
Academic Article rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System.
Academic Article Systemic AAV9-IFN? gene delivery treats highly invasive glioblastoma.
Academic Article Intracranial AAV-IFN-? gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model.
Academic Article In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.
Academic Article Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space.
Academic Article Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.
Academic Article Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis.
Academic Article Adeno-associated virus vector as a platform for gene therapy delivery.
Academic Article CRISPR-Based Therapeutic Genome Editing: Strategies and In?Vivo Delivery by AAV Vectors.
Academic Article AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Academic Article Viral vector platforms within the gene therapy landscape.
Academic Article Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair.
Academic Article Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.
Academic Article Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
Academic Article AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.
Academic Article Durability of transgene expression after rAAV gene therapy.
Academic Article Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery.
Academic Article WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects.
Academic Article Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing.
Academic Article Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor.
Academic Article Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases.
Academic Article Adeno-associated virus as a delivery vector for gene therapy of human diseases.
Academic Article Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus.
Academic Article AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys.
Academic Article Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss.
Academic Article Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders.
Academic Article Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency.
Academic Article The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2.
Academic Article Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review.
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  • Gene therapy