Next-generation AAV vectors for clinical use: an ever-accelerating race

Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c

(2017) Bifeng Pan et al.   NATURE BIOTECHNOLOGY

A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear

(2017) Lukas D Landegger et al.   NATURE BIOTECHNOLOGY

Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction

(2017) Jun Suzuki et al.   Scientific Reports

Adeno-associated Virus (AAV) Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of AAV Serotypes 4, 5, and 11

(2016) Lauriel F. Earley et al.   JOURNAL OF VIROLOGY

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

(2016) Jakob Körbelin et al.   MOLECULAR THERAPY

In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA

(2016) Dustin J Landau et al.   MOLECULAR THERAPY

A novel process of viral vector barcoding and library preparation enables high-diversity library generation and recombination-free paired-end sequencing

(2016) Marcus Davidsson et al.   Scientific Reports

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

AAV ancestral reconstruction library enables selection of broadly infectious viral variants

(2015) J Santiago-Ortiz et al.   GENE THERAPY

E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal—Tailored Acceleration of AAV Evolution

(2015) Dirk Grimm et al.   MOLECULAR THERAPY

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

High-accuracy biodistribution analysis of adeno-associated virus variants by double barcode sequencing

(2015) Damien Marsic et al.   Molecular Therapy-Methods & Clinical Development

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing

(2014) Kei Adachi et al.   Nature Communications

Enhancing the Utility of Adeno-Associated Virus Gene Transfer through Inducible Tissue-Specific Expression

(2013) Shu-Jen Chen et al.   Human Gene Therapy Methods

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

(2012) M A Bartel et al.   GENE THERAPY

Properties of the Adeno-Associated Virus Assembly-Activating Protein

(2012) M. Naumer et al.   JOURNAL OF VIROLOGY

Long-term, efficient inhibition of microRNA function in mice using rAAV vectors

(2012) Jun Xie et al.   NATURE METHODS

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Enhancing the clinical potential of AAV vectors by capsid engineering to evade pre-existing immunity

(2011) Melissa Bartel   Frontiers in Microbiology

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

(2010) A Geisler et al.   GENE THERAPY

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

(2010) C Qiao et al.   GENE THERAPY

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

(2010) F. Sonntag et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

(2008) Wuping Li et al.   MOLECULAR THERAPY

Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic

(2008) Jeffery C Giering et al.   MOLECULAR THERAPY

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

(2008) James T Koerber et al.   MOLECULAR THERAPY