Gene therapy for neurological disorders: progress and prospects

Anti-Amyloid-β Monoclonal Antibodies for Alzheimer’s Disease: Pitfalls and Promise

(2018) Christopher H. van Dyck   BIOLOGICAL PSYCHIATRY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Targeting of nonlipidated, aggregated apoE with antibodies inhibits amyloid accumulation

(2018) Fan Liao et al.   JOURNAL OF CLINICAL INVESTIGATION

Combination anti-Aβ treatment maximizes cognitive recovery and rebalances mTOR signaling in APP mice

(2018) Angie C.A. Chiang et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis

(2018) Andrew E. Arrant et al.   JOURNAL OF NEUROSCIENCE

An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor

(2018) Amanda M. Dudek et al.   JOURNAL OF VIROLOGY

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease

(2018) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

(2018) Kanut Laoharawee et al.   MOLECULAR THERAPY

Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier

(2018) Blake H. Albright et al.   MOLECULAR THERAPY

In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ

(2018) David S. Ojala et al.   MOLECULAR THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Base editing with a Cpf1–cytidine deaminase fusion

(2018) Xiaosa Li et al.   NATURE BIOTECHNOLOGY

Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressiveSOD1ALS

(2018) Michael Benatar et al.   NEUROLOGY

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain

(2018) Yasunori Matsuzaki et al.   NEUROSCIENCE LETTERS

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

(2018) Rachel M. Bailey et al.   Molecular Therapy-Methods & Clinical Development

Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines

(2017) Robert M. Kotin et al.   HUMAN GENE THERAPY

AAV-mediated expression of anti-tau scFvs decreases tau accumulation in a mouse model of tauopathy

(2017) Christina Ising et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Adeno-associated Virus (AAV) Serotypes Have Distinctive Interactions with Domains of the Cellular AAV Receptor

(2017) Sirika Pillay et al.   JOURNAL OF VIROLOGY

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

(2017) Marc Tardieu et al.   LANCET NEUROLOGY

Direct Head-to-Head Evaluation of Recombinant Adeno-associated Viral Vectors Manufactured in Human versus Insect Cells

(2017) Oleksandr Kondratov et al.   MOLECULAR THERAPY

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

(2017) Giuseppe Morabito et al.   MOLECULAR THERAPY

A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

(2017) Maria Grazia Biferi et al.   MOLECULAR THERAPY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Therapeutic Delivery of Butyrylcholinesterase by Brain-Wide Viral Gene Transfer to Mice

(2017) et al.   MOLECULES

ApoE4 markedly exacerbates tau-mediated neurodegeneration in a mouse model of tauopathy

(2017) Yang Shi et al.   NATURE

Enhanced proofreading governs CRISPR–Cas9 targeting accuracy

(2017) Janice S. Chen et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Engineered Cpf1 variants with altered PAM specificities

(2017) Linyi Gao et al.   NATURE BIOTECHNOLOGY

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

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Clinical tests of neurotrophic factors for human neurodegenerative diseases, part 1: Where have we been and what have we learned?

(2017) Raymond T. Bartus et al.   NEUROBIOLOGY OF DISEASE

Global Representations of Goal-Directed Behavior in Distinct Cell Types of Mouse Neocortex

(2017) William E. Allen et al.   NEURON

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

Genome editing abrogates angiogenesis in vivo

(2017) Xionggao Huang et al.   Nature Communications

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS

(2017) Nicholas M. Kanaan et al.   Molecular Therapy-Nucleic Acids

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

(2017) Yoshihide Sehara et al.   Human Gene Therapy Clinical Development

Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome

(2017) Kamal K.E. Gadalla et al.   Molecular Therapy-Methods & Clinical Development

Naturally Occurring Off-Switches for CRISPR-Cas9

(2016) April Pawluk et al.   CELL

A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases

(2016) Jakob Körbelin et al.   EMBO Molecular Medicine

Axonal transport of AAV9 in nonhuman primate brain

(2016) F Green et al.   GENE THERAPY

Therapeutic rAAVrh10 MediatedSOD1Silencing in AdultSOD1G93AMice and Nonhuman Primates

(2016) Florie Borel et al.   HUMAN GENE THERAPY

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

(2016) Michaël Hocquemiller et al.   HUMAN GENE THERAPY

AAV viral vector delivery to the brain by shape-conforming MR-guided infusions

(2016) Krystof S. Bankiewicz et al.   JOURNAL OF CONTROLLED RELEASE

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice

(2016) Wencheng Liu et al.   JOURNAL OF NEUROSCIENCE

Mutants at the 2-Fold Interface of Adeno-associated Virus Type 2 (AAV2) Structural Proteins Suggest a Role in Viral Transcription for AAV Capsids

(2016) Fikret Aydemir et al.   JOURNAL OF VIROLOGY

Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site

(2016) Lin-Ya Huang et al.   JOURNAL OF VIROLOGY

Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders

(2016) Maria Sara Cipolat Mis et al.   MOLECULAR NEUROBIOLOGY

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

(2016) Sourav R Choudhury et al.   MOLECULAR THERAPY

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

(2016) Joshua C Grieger et al.   MOLECULAR THERAPY

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

The antibody aducanumab reduces Aβ plaques in Alzheimer’s disease

(2016) Jeff Sevigny et al.   NATURE

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array

(2016) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

A viral strategy for targeting and manipulating interneurons across vertebrate species

(2016) Jordane Dimidschstein et al.   NATURE NEUROSCIENCE

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

(2016) Lingzhi Zhao et al.   NEUROBIOLOGY OF AGING

Transplantation of spinal cord–derived neural stem cells for ALS

(2016) Jonathan D. Glass et al.   NEUROLOGY

A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

(2016) D. Gowanlock R. Tervo et al.   NEURON

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

rAAV-compatible MiniPromoters for restricted expression in the brain and eye

(2016) Charles N. de Leeuw et al.   Molecular Brain

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

(2016) Jana Miniarikova et al.   Molecular Therapy-Nucleic Acids

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs

(2016) Atsushi Miyanohara et al.   Molecular Therapy-Methods & Clinical Development

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease

(2016) Piotr Hadaczek et al.   Molecular Therapy-Methods & Clinical Development

A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform

(2016) Laura Adamson-Small et al.   Molecular Therapy-Methods & Clinical Development

Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

(2015) Cara M. Weismann et al.   HUMAN MOLECULAR GENETICS

shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model

(2015) Alevtina D. Zharikov et al.   JOURNAL OF CLINICAL INVESTIGATION

Unique Glycan Signatures Regulate Adeno-Associated Virus Tropism in the Developing Brain

(2015) Giridhar Murlidharan et al.   JOURNAL OF VIROLOGY

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers

(2015) Isaac B Hilton et al.   NATURE BIOTECHNOLOGY

Highly efficient Cas9-mediated transcriptional programming

(2015) Alejandro Chavez et al.   NATURE METHODS

Glucocerebrosidase gene therapy prevents α-synucleinopathy of midbrain dopamine neurons

(2015) Emily M. Rocha et al.   NEUROBIOLOGY OF DISEASE

TDP-43 Proteinopathy and ALS: Insights into Disease Mechanisms and Therapeutic Targets

(2015) Emma L. Scotter et al.   Neurotherapeutics

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

(2015) Martin L. Katz et al.   Science Translational Medicine

shRNA mediated knockdown of Nav1.7 in rat dorsal root ganglion attenuates pain following burn injury

(2015) Weihua Cai et al.   BMC Anesthesiology

Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

(2015) Michel Zerah et al.   Human Gene Therapy Clinical Development

Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy

(2014) Marco A. Passini et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotypes 1, 8, and 9 Share Conserved Mechanisms for Anterograde and Retrograde Axonal Transport

(2014) Michael J. Castle et al.   HUMAN GENE THERAPY

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease

(2014) Lisa M. Stanek et al.   HUMAN GENE THERAPY

Delayed Disease Onset and Extended Survival in the SOD1G93A Rat Model of Amyotrophic Lateral Sclerosis after Suppression of Mutant SOD1 in the Motor Cortex

(2014) G. M. Thomsen et al.   JOURNAL OF NEUROSCIENCE

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

(2014) Bin Yang et al.   MOLECULAR THERAPY

Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7

(2014) Pavitra S Ramachandran et al.   MOLECULAR THERAPY

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

(2014) Christian Hinderer et al.   MOLECULAR THERAPY

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

(2014) Morgane Perdomini et al.   NATURE MEDICINE

Huntington disease: natural history, biomarkers and prospects for therapeutics

(2014) Christopher A. Ross et al.   Nature Reviews Neurology

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

(2014) Kei Adachi et al.   Nature Communications

Parvovirus glycan interactions

(2014) Lin-Ya Huang et al.   Current Opinion in Virology

Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons

(2014) Jun-Hyeok Choi et al.   Molecular Brain

Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment

(2014) Olivier Bockstael et al.   Frontiers in Molecular Neuroscience

Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates

(2014) Jonathan B. Rosenberg et al.   Human Gene Therapy Clinical Development

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

(2014) Christian Hinderer et al.   Molecular Therapy-Methods & Clinical Development

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

(2014) Catherine Gérard et al.   Molecular Therapy-Methods & Clinical Development

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

(2013) Lluis Samaranch et al.   HUMAN GENE THERAPY

Intracerebroventricular Injection of Adeno-Associated Virus 6 and 9 Vectors for Cell Type–Specific Transgene Expression in the Spinal Cord

(2013) Elisabeth Dirren et al.   HUMAN GENE THERAPY

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis

(2013) Hongyan Wang et al.   HUMAN MOLECULAR GENETICS

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

(2013) Virginia Haurigot et al.   JOURNAL OF CLINICAL INVESTIGATION

Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery

(2013) Anna Majowicz et al.   JOURNAL OF GENE MEDICINE

Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome

(2013) S. K. Garg et al.   JOURNAL OF NEUROSCIENCE

Adeno-Associated Virus Capsid Proteins May Play a Role in Transcription and Second-Strand Synthesis of Recombinant Genomes

(2013) M. Salganik et al.   JOURNAL OF VIROLOGY

Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice

(2013) Edgardo Rodríguez-Lebrón et al.   MOLECULAR THERAPY

Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

(2013) Kevin D Foust et al.   MOLECULAR THERAPY

AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

(2013) Lluis Samaranch et al.   MOLECULAR THERAPY

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

(2013) Leszek Lisowski et al.   NATURE

Broad protection against influenza infection by vectored immunoprophylaxis in mice

(2013) Alejandro B Balazs et al.   NATURE BIOTECHNOLOGY

RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

(2013) Megan S. Keiser et al.   NEUROBIOLOGY OF DISEASE

An Adeno-Associated Virus-Based Intracellular Sensor of Pathological Nuclear Factor-κB Activation for Disease-Inducible Gene Transfer

(2013) Abdelwahed Chtarto et al.   PLoS One

Glial Promoter Selectivity following AAV-Delivery to the Immature Brain

(2013) Georg von Jonquieres et al.   PLoS One

Tuning gene expression with synthetic upstream open reading frames

(2013) J. P. Ferreira et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection Against Pandemic Influenza

(2013) M. P. Limberis et al.   Science Translational Medicine

A partial MECP2 duplication in a mildly affected adult male: a putative role for the 3' untranslated region in the MECP2 duplication phenotype

(2012) Neil A Hanchard et al.   BMC Medical Genetics

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

(2012) Gabriele Mittermeyer et al.   HUMAN GENE THERAPY

A Mouse Model for MeCP2 Duplication Syndrome: MeCP2 Overexpression Impairs Learning and Memory and Synaptic Transmission

(2012) E. S. Na et al.   JOURNAL OF NEUROSCIENCE

Antibody-Aided Clearance of Extracellular  -Synuclein Prevents Cell-to-Cell Aggregate Transmission

(2012) E.-J. Bae et al.   JOURNAL OF NEUROSCIENCE

Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid

(2012) C. L. Bell et al.   JOURNAL OF VIROLOGY

Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease

(2012) Yong Hong Chen et al.   MOLECULAR THERAPY

Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice

(2012) Kamal KE Gadalla et al.   MOLECULAR THERAPY

Adeno-associated virus (AAV) gene therapy for neurological disease

(2012) Marc S. Weinberg et al.   NEUROPHARMACOLOGY

Clinical and Biomarker Changes in Dominantly Inherited Alzheimer's Disease

(2012) Randall J. Bateman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-Term Follow-Up After Gene Therapy for Canavan Disease

(2012) P. Leone et al.   Science Translational Medicine

An alpha-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons

(2011) Christina E. Khodr et al.   BRAIN RESEARCH

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Corticospinal tract transduction: a comparison of seven adeno-associated viral vector serotypes and a non-integrating lentiviral vector

(2011) T H Hutson et al.   GENE THERAPY

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

(2011) K Varadi et al.   GENE THERAPY

Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs

(2011) T Federici et al.   GENE THERAPY

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

(2011) C Li et al.   GENE THERAPY

Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors

(2011) Steven J. Gray et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

(2011) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

(2011) Peter A LeWitt et al.   LANCET NEUROLOGY

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

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

(2011) Adam K Bevan et al.   MOLECULAR THERAPY

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

Antibody-based protection against HIV infection by vectored immunoprophylaxis

(2011) Alejandro B. Balazs et al.   NATURE

Local and retrograde gene transfer into primate neuronal pathways via adeno-associated virus serotype 8 and 9

(2011) Y. Masamizu et al.   NEUROSCIENCE

T2 Imaging in Monitoring of Intraparenchymal Real-Time Convection-Enhanced Delivery

(2011) R Mark Richardson et al.   NEUROSURGERY

Codon optimization of human factor VIII cDNAs leads to high-level expression

(2010) N. J. Ward et al.   BLOOD

Human Hepatocyte Growth Factor Receptor Is a Cellular Coreceptor for Adeno-Associated Virus Serotype 3

(2010) Chen Ling et al.   HUMAN GENE THERAPY

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

(2010) Elisa Dominguez et al.   HUMAN MOLECULAR GENETICS

Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed

(2010) David B Wang et al.   MOLECULAR THERAPY

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

(2010) Shin-ichi Muramatsu et al.   MOLECULAR THERAPY

Neuroprotection by Gene Therapy Targeting Mutant SOD1 in Individual Pools of Motor Neurons Does not Translate Into Therapeutic Benefit in fALS Mice

(2010) Chris Towne et al.   MOLECULAR THERAPY

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

(2010) Jun Xie et al.   MOLECULAR THERAPY

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Qualitative Imaging of Adeno-Associated Virus Serotype 2–Human Aromatic L-Amino Acid Decarboxylase Gene Therapy in a Phase I Study for the Treatment of Parkinson Disease

(2010) Francisco Valles et al.   NEUROSURGERY

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

(2010) C. F. Valori et al.   Science Translational Medicine

Large-Scale Adeno-Associated Viral Vector Production Using a Herpesvirus-Based System Enables Manufacturing for Clinical Studies

(2009) Nathalie Clément et al.   HUMAN GENE THERAPY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Efficient Gene Delivery and Selective Transduction of Glial Cells in the Mammalian Brain by AAV Serotypes Isolated From Nonhuman Primates

(2009) Patricia A Lawlor et al.   MOLECULAR THERAPY

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

(2009) Eleni A Markakis et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

(2009) A. P. Kells et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Directed evolution of adeno-associated virus to an infectious respiratory virus

(2009) K. J. D. A. Excoffon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

GFAPpromoter elements required for region-specific and astrocyte-specific expression

(2008) Youngjin Lee et al.   GLIA

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

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY

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

(2008) Wuping Li et al.   MOLECULAR THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

(2008) J. L. Eberling et al.   NEUROLOGY

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

(2007) Ronald L Klein et al.   MOLECULAR THERAPY