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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
Administrative Staff
Careers
Resources Links
Labs
Labs
Flotte Lab
Gao Lab
Gray-Edwards Lab
Keeler Lab
Ling Lab
Punzo Lab
Sena-Esteves Lab
Shim Lab
Tai 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
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Viral Vectors
Adeno-Associated Viral (AAV) Vectors
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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
Recombinant Adeno-Associated Virus as a Delivery Platform for Ocular Gene Therapy: A Comprehensive Review
A Comprehensive Atlas of AAV Tropism in the Mouse
Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article
Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders
Engineering a targeted and safe bone anabolic gene therapy to treat osteoporosis in alveolar bone loss
Adeno-associated viral delivery of Env-specific antibodies prevents SIV rebound after discontinuing antiretroviral therapy
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys
Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophy
Oligonucleotide therapies for nonalcoholic steatohepatitis
Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus
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
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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
Ling Lab
Punzo Lab
Sena-Esteves Lab
Shim Lab
Tai 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