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overview

Our research centers around gene therapy using adeno-associated virus (AAV) vectors. We carry out translational gene therapy studies targeting several neurological and metabolic diseases. In addition to targeting specific diseases, we develop therapeutic platforms based on molecular approaches such as genome editing and RNA-based methods. We also develop platform technologies to facilitate AAV-based gene therapy, such as animal modeling and AAV vector production.

In 2020, the lab starts accepting graduate students, postdoctoral researchers, and research associates. Send in your application and get in touch today!

One or more keywords matched the following items that are connected to Wang, Dan
Item TypeName
Concept Gene Expression Profiling
Concept Inhibins
Concept Cytokines
Concept T-Lymphocytes, Cytotoxic
Concept Disease Models, Animal
Concept Fibroblasts
Concept Precision Medicine
Concept Targeted Gene Repair
Concept Inverted Repeat Sequences
Concept Femur
Concept Sequence Analysis, DNA
Concept Spastic Paraplegia, Hereditary
Concept Microscopy, Electron
Concept Bone Resorption
Concept Blood-Brain Barrier
Concept Humans
Concept Sequence Analysis, RNA
Concept Animals, Newborn
Concept Prostatic Neoplasms
Concept Heterozygote
Concept Genetic Therapy
Concept Embryo, Mammalian
Concept Macaca fascicularis
Concept Magnetic Resonance Imaging
Concept Gene Expression
Concept Rare Diseases
Concept Mutagenesis, Site-Directed
Concept Cats
Concept RNA Interference
Concept Mice, Inbred BALB C
Concept Body Weight
Concept CD8-Positive T-Lymphocytes
Concept RNA Editing
Concept Amyotrophic Lateral Sclerosis
Concept Hereditary Sensory and Autonomic Neuropathies
Concept Injections
Concept Dependovirus
Concept Endonucleases
Concept Osteoblasts
Concept Lysosomes
Concept Population Control
Concept Glial Fibrillary Acidic Protein
Concept Tropism
Concept Ovarian Follicle
Concept Dendritic Cells
Concept Protein Biosynthesis
Concept Ovarian Neoplasms
Concept Mice, Knockout
Concept Antineoplastic Agents
Concept Injections, Spinal
Concept Infusion Pumps, Implantable
Concept Designer Drugs
Concept Heart Failure
Concept Adenomatous Polyposis Coli Protein
Concept Frontotemporal Dementia
Concept Fallopian Tubes
Concept Cystic Fibrosis Transmembrane Conductance Regulator
Concept Protein Synthesis Inhibitors
Concept Mice
Concept Clinical Trials as Topic
Concept Epilepsy
Concept Blastocyst
Concept Transcription, Genetic
Concept Iduronidase
Concept Cattle
Concept Transgenes
Concept Mice, Inbred C57BL
Concept Congenital Disorders of Glycosylation
Concept DNA Repeat Expansion
Concept Sequence Deletion
Concept Prostate
Concept Mutation
Concept Cells, Cultured
Concept Brain Mapping
Concept Mice, Transgenic
Concept Oxygen
Concept Capsid
Concept Adrenoleukodystrophy
Concept Nucleic Acid Conformation
Concept Xenograft Model Antitumor Assays
Concept Anti-Mullerian Hormone
Concept Cell Proliferation
Concept Homeodomain Proteins
Concept Ganglia, Spinal
Concept alpha 1-Antitrypsin
Concept DNA, Viral
Concept Genetic Diseases, Inborn
Concept Animals, Wild
Concept Codon, Terminator
Concept Genetic Engineering
Concept Macaca mulatta
Concept Absorptiometry, Photon
Concept Tyrosinemias
Concept Genes, Reporter
Concept Primary Ovarian Insufficiency
Concept Gene Expression Regulation, Enzymologic
Concept Cerebrospinal Fluid
Concept Survival Analysis
Concept Green Fluorescent Proteins
Concept Gene Order
Concept Reverse Transcriptase Polymerase Chain Reaction
Concept Recombinant Fusion Proteins
Concept Oxadiazoles
Concept Lymphocyte Activation
Concept Cerebrovascular Circulation
Concept Maple Syrup Urine Disease
Concept Dipeptides
Concept Transduction, Genetic
Concept Spheroids, Cellular
Concept Adenoviridae
Concept Muscular Atrophy, Spinal
Concept Hemodynamics
Concept Mice, Inbred Strains
Concept Microfilament Proteins
Concept Reproducibility of Results
Concept Ovary
Concept Granulosa Cells
Concept Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase
Concept Mucopolysaccharidosis II
Concept Antigen-Presenting Cells
Concept Rats
Concept alpha 1-Antitrypsin Deficiency
Concept Spleen
Concept Mucopolysaccharidosis I
Concept Codon, Nonsense
Concept DNA Replication
Concept Proteomics
Concept RNA Splicing
Concept Cell Line
Concept Contraceptive Agents
Concept Rats, Sprague-Dawley
Concept Ovalbumin
Concept Serotyping
Concept United States Food and Drug Administration
Concept Gene Deletion
Concept Genetic Vectors
Concept Uterus
Concept Gangliosides
Concept MicroRNAs
Concept Lumbar Vertebrae
Concept Visual Cortex
Concept Gene Silencing
Concept Base Sequence
Concept Alleles
Concept Plasmids
Concept Genes, Recessive
Concept Gene Knockdown Techniques
Concept Embryonic Development
Concept 3-Methyl-2-Oxobutanoate Dehydrogenase (Lipoamide)
Concept Genome, Viral
Concept Gene Transfer Techniques
Concept Cell Line, Tumor
Concept Molecular Sequence Data
Concept Osmotic Pressure
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 Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.
Academic Article Adeno-associated Virus Serotype Vectors Efficiently Transduce Normal Prostate Tissue and Prostate Cancer Cells.
Academic Article Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity.
Academic Article A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.
Academic Article Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.
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 Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses.
Academic Article Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS.
Academic Article A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord.
Academic Article Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppression.
Academic Article Suppression of premature termination codons as a therapeutic approach.
Academic Article The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse.
Academic Article Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.
Academic Article Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB.
Academic Article AMH/MIS as a contraceptive that protects the ovarian reserve during chemotherapy.
Academic Article CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector.
Academic Article AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer.
Academic Article Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy.
Academic Article Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.
Academic Article Adeno-associated virus vector as a platform for gene therapy delivery.
Academic Article Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity.
Academic Article Single-cell sequencing of neonatal uterus reveals an Misr2+ endometrial progenitor indispensable for fertility.
Academic Article Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis.
Academic Article Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus.
Academic Article CRISPR-Based Therapeutic Genome Editing: Strategies and In?Vivo Delivery by AAV Vectors.
Academic Article Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy.
Academic Article AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity.
Academic Article AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies.
Academic Article Single-cell sequencing reveals suppressive transcriptional programs regulated by MIS/AMH in neonatal ovaries.
Academic Article Juggling Safety and Efficacy: Finding Ways to Achieve Both.
Academic Article AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.
Academic Article Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.
Concept Neurovascular Coupling
Concept Cerebral Blood Volume
Concept Gene Editing
Concept HEK293 Cells
Concept Infusions, Intraventricular
Concept Single-Cell Analysis
Concept Nonsense Mediated mRNA Decay
Concept Non-alcoholic Fatty Liver Disease
Concept DNA End-Joining Repair
Concept Recombinational DNA Repair
Concept Infusions, Spinal
Concept Clustered Regularly Interspaced Short Palindromic Repeats
Concept CRISPR-Cas Systems
Concept Ovarian Reserve
Concept Gray Matter
Academic Article AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.
Academic Article Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Academic Article Ataluren suppresses a premature termination codon in an MPS I-H mouse.
Academic Article Rescue of GM3 synthase deficiency by spatially controlled, rAAV-mediated ST3GAL5 delivery.
Academic Article Base editing rescue of spinal muscular atrophy in cells and in mice.
Academic Article Durable contraception in the female domestic cat using viral-vectored delivery of a feline anti-M?llerian hormone transgene.
Academic Article C9orf72 poly(PR) aggregation in nucleus induces ALS/FTD-related neurodegeneration in cynomolgus monkeys.
Academic Article Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice.
Academic Article Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection.
Academic Article AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys.
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 A comprehensive atlas of AAV tropism in the mouse.
Academic Article Dynamics of the cerebral blood flow response to brief neural activity in human visual cortex.
Academic Article BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
Academic Article Gene therapy then and now: A look back at changes in the field over the past 25 years.
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