PerURLs

AAV Capsid Engineering✕

Precision glycoengineered AAV capsids enhance hepatocyte targeting and attenuated immune activation for liver-directed gene therapy. Biomaterials

AI-engineered AAV capsid enables intravitreal delivery for the treatment of diverse retinal degenerations. Molecular therapy. Advances

Optimization of AAV tools to target Müller glial cells for retinal gene therapy. Molecular therapy. Advances

Systematic evaluation of BBB-penetrant AAV capsids in marmosets identifies VCAP-102 as a highly efficient brain-transducing capsid. Molecular therapy. Advances

Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retina. Molecular therapy. Advances

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice nature.com

AAV Immunogenicity And Antibodies✕

Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency. Molecular therapy. Advances

Adeno-Associated Virus Gene Therapy for Spinal Muscular Atrophy Induces Hepatotoxicity via Cytokine and Macrophage Activation. Liver international : official journal of the International Association for the Study of the Liver

AAV Manufacturing And Analytics✕

Anion-exchange chromatography separates structurally heterogeneous and low-potency particles in adeno-associated virus manufacture. Molecular therapy. Advances

AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality. Molecular therapy. Advances

Omics-guided engineering of CHO cells reveals host targets to support AAV production. Molecular therapy. Advances

Erratum: Impact of downstream purification process and AAV serotype on protein-impurity clearance. Molecular therapy. Advances

CRISPR-Cas9 screening reveals TM9SF2 knockout as a solution to HEK293 cell aggregation for improved AAV production. Molecular therapy. Advances

It's what's on the inside that counts: Capsid content as a critical quality attribute for AAV characterization. Molecular therapy. Advances

Challenges and opportunities in continuous bioprocessing of lentiviral vectors and adeno-associated viral vectors. Biotechnology advances

Optimizing LOD/LOQ and sample consumption during analytical ultracentrifugation to characterize AAV gene therapy vectors. Journal of pharmaceutical and biomedical analysis

The evolving landscape of recombinant adeno-associated virus (rAAV) manufacturing: Engineering vector plasmids for upstream production. Biotechnology advances

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production. Gene therapy

Advanced spent media analytics and multivariate data analysis for AAV production media optimization. Journal of chromatography. A

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies nature.com

AAV Delivery And Biology✕

Serotype-dependent differences in AAV cellular transduction rates in the hypothalamus of Arctic ground squirrels. Journal of neuroscience methods

Whole-brain biodistribution of AAV2, AAV8, and AAV-B10 after unilateral intracerebroventricular injection. Molecular therapy. Advances

Intracellular trafficking and nuclear compartmentalization of AAV gene delivery. Molecular therapy. Advances

Comparative evaluation of adeno-associated virus and lentivirus mediated gene transfer in adult rat optic nerve. Experimental eye research

Suppression of AAV-Delivered Transgene Expression Using Artificial MicroRNAs Delivered by an Alternative AAV Serotype. Human gene therapy

Small RNA Barcodes for Sensitive Labeling of AAV Transcripts. eNeuro

Evaluating cases of possible insertional mutagenesis after AAV gene therapy. Molecular therapy : the journal of the American Society of Gene Therapy

Hybrid AAV-LNP transposition system lowers the therapeutic threshold and improves transgene stability for liver-directed gene therapy. Molecular therapy : the journal of the American Society of Gene Therapy

Construct optimization for AAV-mediated human α-syn overexpression, and validation across research settings: Development of a shared tool for the research community. Journal of Parkinson's disease

AAV Therapeutic Applications✕

Could AAV-vectored delivery of broadly neutralizing antibodies (bNAbs) reduce virologic failure in children diagnosed with perinatal HIV? Human vaccines & immunotherapeutics

Erratum: Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness. Molecular therapy. Advances

Highly potent MyoAAV4A vector reverses GSD III pathology in aged mice and enables long-term muscle disease correction in young adult mice. Molecular therapy. Advances

Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency. Molecular therapy. Advances

AAV-norrin gene therapy rescues retinal defects in mice with Norrie disease and oxygen-induced retinopathy. Molecular therapy. Advances

First pilot study of intrastromal rAAV-PAX6 gene therapy suggests improved corneal thickness and transcription correction in aniridic mouse. Molecular therapy. Advances

Defining the safety and efficacy of liver-directed AAV gene therapy using a human liver tissue-equivalent platform. Molecular therapy. Advances

Aquaporin-1 gene therapy as a novel method to treat radiation-induced salivary gland damage: The case for adeno-associated virus vectors. Oral oncology

Efficacy and safety of SENS-501, a dual-AAV otoferlin gene therapy, for DFNB9 congenital deafness. Molecular therapy. Advances

Preclinical evaluation of AAV2 anti-VEGF gene therapy using scFv for choroidal neovascularization. Molecular therapy. Advances

Adeno-Associated virus-based approaches for mitochondrial diseases: advances and challenges. Molecular psychiatry

Long-term depletion of KIR3DL01+ NK cells by adeno-associated viral-vectored antibody delivery alters chronic SIV infection. PLoS pathogens

A self-complementary recombinant adeno-associated virus vector coding for an anchorless prion protein carrying the G127V mutation extends survival in a rodent prion disease model. PLoS pathogens

Adeno-Associated Virus-Mediated Central Nervous System Gene Transfer to Suppress Alzheimer's Disease High-Risk APOE4 Variant and Replace with Protective APOE2. Human gene therapy

The efficacy and safety of adeno-associated virus-mediated gene therapy for CNGA3- and CNGB3-associated achromatopsia: A genotype-aware systematic review and meta-analysis. Survey of ophthalmology

Five-Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study. Muscle & nerve

AAV-mediated FGF21 gene therapy promotes health span extension by whole-body tissue-specific adaptations. Molecular therapy : the journal of the American Society of Gene Therapy