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The Horae (红瑞) Gene Therapy Center
About
About
Horae GTC Center
Faculty
Faculty Publications
Faculty Publications
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Affiliated Faculty
Affiliates
Affiliates
Bioinformatics
RNA Therapeutics Institute (RTI)
Center for Clinical Translational Science
Sherman Center
China Program Office
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Flotte Lab
Gao Lab
Gray-Edwards Lab
Keeler Lab
Punzo Lab
Sena-Esteves Lab
Zhong Lab
Gene Therapy
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Research
Alpha-One Antitrypsin Deficiency
Amyotrophic Lateral Sclerosis (ALS)
Canavan Disease
Ciliopathies
Cystic Fibrosis
Fatty Acid Oxidation Deficiencies, VLCAD
Huntington Disease
Hypercholesterimia
Pompe Disease
Retinitis Pigmentosa
Tay-Sachs Disease
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What is a Clinical Trial
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Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Guangping Gao, PhD
Guangping Gao, PhD
Visit Lab Website
Contact Dr. Gao
Total:
50
results
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
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About
About
Horae GTC Center
Faculty
Faculty Publications
Faculty Publications
Terence Flotte, MD
Guangping Gao, PhD
Claudio Punzo, PhD
Miguel Sena-Esteves, PhD
Neil Aronin, MD
Robert Brown, D.Phil, MD
Affiliated Faculty
Affiliates
Affiliates
Bioinformatics
RNA Therapeutics Institute (RTI)
Center for Clinical Translational Science
Sherman Center
China Program Office
Administrative Staff
Careers
Resources Links
Labs
Labs
Flotte Lab
Gao Lab
Gray-Edwards Lab
Keeler Lab
Punzo Lab
Sena-Esteves Lab
Zhong Lab
Gene Therapy
Research
Research
Alpha-One Antitrypsin Deficiency
Amyotrophic Lateral Sclerosis (ALS)
Canavan Disease
Ciliopathies
Cystic Fibrosis
Fatty Acid Oxidation Deficiencies, VLCAD
Huntington Disease
Hypercholesterimia
Pompe Disease
Retinitis Pigmentosa
Tay-Sachs Disease
Clinical Trials
Clinical Trials
What is a Clinical Trial
Clinical Trials at UMass
Clinical Trial Resources
Viral Vectors
Viral Vectors
Adeno-Associated Viral (AAV) Vectors
Viral Vector Core
IBC-Related Facts
News & Events
News & Events
Gene Therapy News
Past Events & Videos
GTC Labs Blog Posts
Partner with Us
Contact
Contact
Contact Information
For Media
Donate
Additional Resources