Item Type | Name |
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.
|