Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders

Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin

(2021) Pike-See Cheah et al.   Science Advances

Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

(2021) Meaghan Van Alstyne et al.   NATURE NEUROSCIENCE

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

(2021) Jerry R. Mendell et al.   JAMA Neurology

Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

(2021) Ana Fajardo-Serrano et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Highly accurate protein structure prediction with AlphaFold

(2021) John Jumper et al.   NATURE

Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?

(2020) Patrizia Tornabene et al.   HUMAN GENE THERAPY

The 3′ Untranslated Region Protects the Heart from Angiotensin II-Induced Cardiac Dysfunction via AGGF1 Expression

(2020) Lexi Ding et al.   MOLECULAR THERAPY

An expanded library of orthogonal split inteins enables modular multi-peptide assemblies

(2020) Filipe Pinto et al.   Nature Communications

Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters

(2020) Bart Nieuwenhuis et al.   GENE THERAPY

Assessment of AAV Dual Vector Safety in the Abca4−/− Mouse Model of Stargardt Disease

(2020) Michelle E. McClements et al.   Translational Vision Science & Technology

CNS transduction benefits of AAV-PHP.eB over AAV9 are dependent on administration route and mouse strain.

(2020) Sophie N. Mathiesen et al.   Molecular Therapy-Methods & Clinical Development

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model

(2019) Omar Akil et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Feasibility of using NF1-GRD and AAV for gene replacement therapy in NF1-associated tumors

(2019) Ren-Yuan Bai et al.   GENE THERAPY

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

(2019) Patrizia Tornabene et al.   Science Translational Medicine

Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

(2019) Trapani   Genes

Onasemnogene Abeparvovec: First Global Approval

(2019) Sheridan M. Hoy   DRUGS

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease

(2019) Frank M. Dyka et al.   HUMAN GENE THERAPY

Dual-AAV delivery of large gene sequences to the inner ear

(2019) Ellen Reisinger   HEARING RESEARCH

Structural puzzles in virology solved with an overarching icosahedral design principle

(2019) Reidun Twarock et al.   Nature Communications

Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

(2018) Wei Zhang et al.   HUMAN GENE THERAPY

Recent progress and considerations for AAV gene therapies targeting the central nervous system

(2018) Erik Allen Lykken et al.   Journal of Neurodevelopmental Disorders

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Triple Vectors Expand AAV Transfer Capacity in the Retina

(2018) Andrea Maddalena et al.   MOLECULAR THERAPY

Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin

(2018) Chunping Qiao et al.   Molecular Therapy-Methods & Clinical Development

A dual‐AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock‐out mice

(2018) Hanan Al‐Moyed et al.   EMBO Molecular Medicine

An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4−/− Mice

(2018) Michelle E. McClements et al.   HUMAN GENE THERAPY

AAV Immunogenicity: New Answers Create New Questions

(2018) Jamie L. Shirley et al.   MOLECULAR THERAPY

A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration

(2018) Suddhasil Mookherjee et al.   Cell Reports

Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

(2018) Kousaku Ohno et al.   Molecular Therapy-Methods & Clinical Development

Gene therapy for blood diseases

(2018) Donald B Kohn   CURRENT OPINION IN BIOTECHNOLOGY

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

(2017) Diane Golebiowski et al.   HUMAN GENE THERAPY

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

(2017) Eric R. Pozsgai et al.   MOLECULAR THERAPY

Radically truncated MeCP2 rescues Rett syndrome-like neurological defects

(2017) Rebekah Tillotson et al.   NATURE

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain

(2017) Jun Nishiyama et al.   NEURON

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting

(2017) Livia S. Carvalho et al.   Frontiers in Neuroscience

Evolutionary Insights into RNA trans-Splicing in Vertebrates

(2016) Quan Lei et al.   Genome Biology and Evolution

253. Expanded Packaging Capacity of AAV by Lumenal Charge Alteration

(2016) Matthew Tiffany et al.   MOLECULAR THERAPY

Gene therapies: the challenge of super-high-cost treatments and how to pay for them

(2016) David R Carr et al.   Regenerative Medicine

Split-inteins and their bioapplications

(2015) Yifeng Li   BIOTECHNOLOGY LETTERS

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

(2015) Ivana Trapani et al.   HUMAN MOLECULAR GENETICS

Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice

(2015) F Jason Duncan et al.   MOLECULAR THERAPY

Adeno-associated Vector Toxicity—To Be or Not to Be?

(2015) Hildegard Büning et al.   MOLECULAR THERAPY

A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence

(2015) Marina Pryadkina et al.   Molecular Therapy-Methods & Clinical Development

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

(2014) P Colella et al.   GENE THERAPY

Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors

(2014) William Lostal et al.   HUMAN GENE THERAPY

Nature's recipe for splitting inteins

(2014) A. S. Aranko et al.   PROTEIN ENGINEERING DESIGN & SELECTION

More efficient repair of DNA double-strand breaks in skeletal muscle stem cells compared to their committed progeny

(2014) Leyla Vahidi Ferdousi et al.   Stem Cell Research

Astrocytic modulation of blood brain barrier: perspectives on Parkinson’s disease

(2014) Ricardo Cabezas et al.   Frontiers in Cellular Neuroscience

Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A

(2014) Frank M. Dyka et al.   Human Gene Therapy Methods

Recent advances in in vivo applications of intein-mediated protein splicing

(2014) Natalya I Topilina et al.   Mobile DNA

Inflammatory and immune responses in the cochlea: potential therapeutic targets for sensorineural hearing loss

(2014) Masato Fujioka et al.   Frontiers in Pharmacology

DNA repair mechanisms in dividing and non-dividing cells

(2013) Teruaki Iyama et al.   DNA REPAIR

Effective delivery of large genes to the retina by dual AAV vectors

(2013) Ivana Trapani et al.   EMBO Molecular Medicine

Adeno-associated virus structural biology as a tool in vector development

(2013) Lauren M Drouin et al.   Future Virology

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

(2013) V S Lopes et al.   GENE THERAPY

Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery

(2013) A B Katwal et al.   GENE THERAPY

Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

(2013) Yadong Zhang et al.   HUMAN MOLECULAR GENETICS

Extein Residues Play an Intimate Role in the Rate-Limiting Step of Protein Trans-Splicing

(2013) Neel H. Shah et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy

(2013) Pasqualina Colella et al.   PLoS One

Inteins: nature's gift to protein chemists

(2013) Neel H. Shah et al.   Chemical Science

Alstrom Syndrome: Genetics and Clinical Overview

(2012) Jan D. Marshall et al.   CURRENT GENOMICS

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

(2012) M. A. DiMattia et al.   JOURNAL OF VIROLOGY

Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

(2012) William E. Grose et al.   PLoS One

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

(2011) Yadong Zhang et al.   HUMAN GENE THERAPY

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Efficient Transgene Reconstitution with Hybrid Dual AAV Vectors Carrying the Minimized Bridging Sequences

(2010) Arkasubhra Ghosh et al.   HUMAN GENE THERAPY

Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

(2010) Matthew L Hirsch et al.   MOLECULAR THERAPY

Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6

(2010) Guy L Odom et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Astrocyte function and role in motor neuron disease: A future therapeutic target?

(2009) Daniel Blackburn et al.   GLIA

Frequency and phenotype of SPG11 and SPG15 in complicated hereditary spastic paraplegia

(2009) R Schule et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

(2009) Yi Lai et al.   MOLECULAR THERAPY

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

(2009) Biao Dong et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

(2008) Mariacarmela Allocca et al.   JOURNAL OF CLINICAL INVESTIGATION

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Targeted Integration of Functional Human ATM cDNA Into Genome Mediated by HSV/AAV Hybrid Amplicon Vector

(2007) Maria L Cortés et al.   MOLECULAR THERAPY

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY