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Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution ScienceDirect.com

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B The New England Journal of Medicine

US Pharmacopeia Recognizes Mass Photometry in New AAV Reference Standards for Gene Therapy Quality Genetic Engineering and Biotechnology News

Spatial genomics of AAV vectors reveals mechanism of transcriptional crosstalk that enables targeted delivery of large genetic cargo Nature

Mass photometry recognized by U.S. Pharmacopeia in new AAV reference standards for gene therapy quality News-Medical

Potassium KCa3.1 channel overexpression deteriorates functionality and availability of channels at the outer cellular membrane Nature

Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat PNAS

AAV-mediated gene therapy: Advancing cardiovascular disease treatment Frontiers

An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery Science | AAAS

Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier ScienceDirect.com

Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings Frontiers

Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain Nature

Innate immune response to AAV-based gene therapy vectors: mechanisms of complement activation and cytokine release ScienceDirect.com

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

Optimization of AAV vectors for transactivator-regulated enhanced gene expression within targeted neuronal populations ScienceDirect.com

AAV vectors: The Rubik’s cube of human gene therapy ScienceDirect.com

Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models Nature

An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction ScienceDirect.com

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy Nature

Heterogeneity, a weak link between constrained AAV dose and long-term efficacy of gene therapy to muscular dystrophy: A critical and prospective review ScienceDirect.com

Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters | Gene Therapy Nature

Adeno-Associated Viruses (AAV) and Host Immunity – A Race Between the Hare and the Hedgehog Frontiers

Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing Nature

T Cell-Mediated Immune Responses to AAV and AAV Vectors Frontiers

AAV-mediated co-expression of an immunogenic transgene plus PD-L1 enables sustained expression through immunological evasion Nature

Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications ScienceDirect.com

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations ScienceDirect.com

Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease ScienceDirect.com

Deep Parallel Characterization of AAV Tropism and AAV-Mediated Transcriptional Changes via Single-Cell RNA Sequencing Frontiers

Intravitreal AAV-Delivery of Genetically Encoded Sensors Enabling Simultaneous Two-Photon Imaging and Electrophysiology of Optic Nerve Axons Frontiers

AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset Nature

Optimization of AAV vectors to target persistent viral reservoirs Virology Journal

Prolonged Trapping of Adeno-Associated Virus Capsids Reveals that Genome Packaging Affects Single-Ion Mass Spectrometry Measurements ACS Publications

Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency ScienceDirect.com

SERCA2a overexpression improves muscle function in a canine Duchenne muscular dystrophy model ScienceDirect.com

Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease Nature

Empty and Full AAV Capsid Charge and Hydrophobicity Differences Measured with Single-Particle AFM ACS Publications

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy Science | AAAS

AAV-mediated upregulation of VDAC1 rescues the mitochondrial respiration and sirtuins expression in a SOD1 mouse model of inherited ALS Nature

Separation and analysis of empty and filled capsids during viral vector production Labiotech.eu

Unleashing the potential of AAV gene therapy Nature

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction ScienceDirect.com

Long-Term Delivery of an Anti-SIV Monoclonal Antibody With AAV Frontiers

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B The New England Journal of Medicine

Standard screening methods underreport AAV-mediated transduction and gene editing Nature

Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B The New England Journal of Medicine

Synthetic Biology: Emerging Concepts to Design and Advance Adeno-Associated Viral Vectors for Gene Therapy Wiley Online Library

Nitrous oxide activates layer 5 prefrontal neurons via SK2 channel inhibition for antidepressant effect Nature

Sania Therapeutics presents latest from AAV gene therapy GlobeNewswire

Dopamine drives neuronal excitability via KCNQ channel phosphorylation for reward behavior ScienceDirect.com

In vivo dynamics of AAV-mediated gene delivery to sensory neurons of the trigeminal ganglia Nature

Ectopic expression of a mechanosensitive channel confers spatiotemporal resolution to ultrasound stimulations of neurons for visual restoration Nature

Adipose-specific deletion of the cation channel TRPM7 inhibits TAK1 kinase-dependent inflammation and obesity in male mice Nature

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A The New England Journal of Medicine

Epilepsy Gene Therapy Using an Engineered Potassium Channel Journal of Neuroscience

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B The New England Journal of Medicine

Improved calcium sensor GCaMP-X overcomes the calcium channel perturbations induced by the calmodulin in GCaMP Nature