Guangping Gao, PhD

• AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva

• Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

• Therapeutic inhibition of miR-155 attenuates liver fibrosis via STAT3 signaling

• Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing

• C9orf72 poly(PR) aggregation in nucleus induces ALS/FTD-related neurodegeneration in cynomolgus monkeys

• Durable contraception in the female domestic cat using viral-vectored delivery of a feline anti-Müllerian hormone transgene

• A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors

• Renewal of oligodendrocyte lineage reverses dysmyelination and CNS neurodegeneration through corrected N-acetylaspartate metabolism

• Schnurri-3 inhibition suppresses bone and joint damage in models of rheumatoid arthritis

• Engineering Nme2Cas9 Adenine Base Editors with Improved Activity and Targeting Scope

• Rescue of GM3 synthase deficiency by spatially controlled, rAAV-mediated ST3GAL5 delivery

• Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

• Base editing rescue of spinal muscular atrophy in cells and in mice

• Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo

• Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection

• Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases

• Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration

• Letter to the Editor: mRNA Vaccines in Urological Malignancies

• Phosphorylation of RXRα mediates the effect of JNK to suppress hepatic FGF21 expression and promote metabolic syndrome

• Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery

• WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects

• Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes

• Ocular Drug Delivery: Advancements and Innovations

• Paradoxical activation of transcription factor SREBP1c and de novo lipogenesis by hepatocyte-selective ATP-citrate lyase depletion in obese mice

• AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy

• Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector

• Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

• Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

• Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles

• Graphene quantum dots rescue angiogenic retinopathy via blocking STAT3/Periostin/ERK signaling

• AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

• Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity

• Durability of transgene expression after rAAV gene therapy

• Regulation of sclerostin by the SIRT1 stabilization pathway in osteocytes

• High concordance of ELISA and neutralization assays allows for the detection of antibodies to individual AAV serotypes

• Proteomic changes of aqueous humor in proliferative diabetic retinopathy patients treated with different intravitreal anti-VEGF agents

• Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics"

• rAAV-delivered PTEN therapeutics for prostate cancer

• Membranous Bubbles: High-Purity and High-Titer Exosomes as the Potential Solution for Adeno-Associated Viruses to Evade Neutralization?

• Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

• Dynamics of a disinhibitory prefrontal microcircuit in controlling social competition

• Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

• AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway

• Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

• Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary

• Cellular and Tissue Selectivity of AAV Serotypes for Gene Delivery to Chondrocytes and Cartilage

• Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics

• Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency

• Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword

• rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression