Guangping Gao, PhD

• Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency

• Adeno-associated virus as a delivery vector for gene therapy of human diseases

• Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection

• Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases

• Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

• Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope

• AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta

• Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis

• Aurora kinase B disruption suppresses pathological retinal angiogenesis by affecting cell cycle progression

• A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors

• Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo

• NIIMBL's viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing

• Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice

• 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"