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

[1]  Elena A. Westeinde,et al.  The gut commensal Blautia maintains colonic mucus function under low-fiber consumption through secretion of short-chain fatty acids , 2022, bioRxiv.

[2]  Garreck H. Lenz,et al.  Enhancer viruses for combinatorial cell-subclass-specific labeling , 2021, Neuron.

[3]  Mingyao Li,et al.  MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates , 2020, Science Translational Medicine.

[4]  David Kulp,et al.  Innovations present in the primate interneuron repertoire , 2020, Nature.

[5]  Tom P. Franken,et al.  Viral manipulation of functionally distinct interneurons in mice, non-human primates and humans , 2020, Nature Neuroscience.

[6]  G. Ronzitti,et al.  Human Immune Responses to Adeno-Associated Virus (AAV) Vectors , 2020, Frontiers in Immunology.

[7]  Sripriya Ravindra Kumar,et al.  Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types , 2020, Nature Methods.

[8]  F. Mingozzi,et al.  AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer. , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[9]  J. Mendell,et al.  From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1. , 2019, Pediatric neurology.

[10]  Concha Bielza,et al.  A community-based transcriptomics classification and nomenclature of neocortical cell types , 2019, Nature Neuroscience.

[11]  V. Gradinaru,et al.  TRIM9-Mediated Resolution of Neuroinflammation Confers Neuroprotectionupon Ischemic Stroke in Mice , 2019, Cell reports.

[12]  M. Mortrud,et al.  Functional enhancer elements drive subclass-selective expression from mouse to primate neocortex , 2019, bioRxiv.

[13]  Yuna Sun,et al.  Adeno-associated virus 2 bound to its cellular receptor AAVR , 2019, Nature Microbiology.

[14]  Sripriya Ravindra Kumar,et al.  Systemic AAV vectors for widespread and targeted gene delivery in rodents , 2019, Nature Protocols.

[15]  F. Piguet,et al.  Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia. , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[16]  R. Klein,et al.  More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B , 2018, Gene Therapy.

[17]  H. Gritton,et al.  A MicroRNA-Based Gene-Targeting Tool for Virally Labeling Interneurons in the Rodent Cortex. , 2018, Cell reports.

[18]  David J. Anderson,et al.  The Neuropeptide Tac2 Controls a Distributed Brain State Induced by Chronic Social Isolation Stress , 2018, Cell.

[19]  H. Evens,et al.  Haemophilia gene therapy: From trailblazer to gamechanger , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[20]  I. Alexander,et al.  Gene therapy clinical trials worldwide to 2017: An update , 2018, The journal of gene medicine.

[21]  P. Kubes,et al.  Immune Responses in the Liver. , 2018, Annual review of immunology.

[22]  M. Y. Rincon,et al.  Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector , 2018, Gene Therapy.

[23]  Qiang Wang,et al.  The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice. , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[24]  G. Demontis,et al.  Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery , 2018, Human molecular genetics.

[25]  H. Ameri.  Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation , 2018, Journal of current ophthalmology.

[26]  T. Flotte,et al.  Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First. , 2018, Human gene therapy.

[27]  Yukihiro Okada,et al.  Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain , 2018, Neuroscience Letters.

[28]  James M. Wilson,et al.  Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN. , 2018, Human gene therapy.

[29]  B. Roska,et al.  Virus stamping for targeted single-cell infection in vitro and in vivo , 2017, Nature Biotechnology.

[30]  L. Leocani,et al.  AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[31]  W. Strohl,et al.  Adeno-Associated Virus (AAV) as a Vector for Gene Therapy , 2017, BioDrugs.

[32]  Douglas R Martin,et al.  Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain. , 2017, Human gene therapy.

[33]  V. Gradinaru,et al.  Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems , 2017, Nature Neuroscience.

[34]  William E. Allen,et al.  Global Representations of Goal-Directed Behavior in Distinct Cell Types of Mouse Neocortex , 2017, Neuron.

[35]  Andreas Hierlemann,et al.  Causal evidence for retina dependent and independent visual motion computations in mouse cortex , 2017, Nature Neuroscience.

[36]  Arun Srivastava,et al.  In vivo tissue-tropism of adeno-associated viral vectors. , 2016, Current opinion in virology.

[37]  S. Parker,et al.  Adeno-Associated Virus-Based Gene Therapy for CNS Diseases , 2016, Human gene therapy.

[38]  B. Gao,et al.  Basic liver immunology , 2016, Cellular & Molecular Immunology.

[39]  R. Samulski,et al.  AAVR: A Multi-Serotype Receptor for AAV. , 2016, Molecular therapy : the journal of the American Society of Gene Therapy.

[40]  Ken Sugino,et al.  A Mammalian enhancer trap resource for discovering and manipulating neuronal cell types , 2016, eLife.

[41]  Sripriya Ravindra Kumar,et al.  Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain , 2015, Nature Biotechnology.

[42]  Steven J. M. Jones,et al.  Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors , 2014, Molecular therapy. Methods & clinical development.

[43]  K. High,et al.  Immune responses to AAV vectors: overcoming barriers to successful gene therapy. , 2013, Blood.

[44]  D. Gaudet,et al.  Gene therapy for lipoprotein lipase deficiency , 2012, Current opinion in lipidology.

[45]  J. Prieto,et al.  Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates , 2012, Journal of Translational Medicine.

[46]  B. Byrne,et al.  Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9 , 2012, Journal of Virology.

[47]  P. Aebischer,et al.  Parkinson's disease: gene therapies. , 2012, Cold Spring Harbor perspectives in medicine.

[48]  K. Foust,et al.  Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. , 2012, Human gene therapy.

[49]  J. Kleinschmidt,et al.  Peptide Ligands Incorporated into the Threefold Spike Capsid Domain to Re-Direct Gene Transduction of AAV8 and AAV9 In Vivo , 2011, PloS one.

[50]  M. Ehlers,et al.  Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. , 2011, Human gene therapy.

[51]  S. Randell,et al.  Terminal N-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9* , 2011, The Journal of Biological Chemistry.

[52]  R. Samulski,et al.  Viral vectors and delivery strategies for CNS gene therapy. , 2010, Therapeutic delivery.

[53]  Lili Wang,et al.  Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. , 2009, Human gene therapy.

[54]  J. Rabinowitz,et al.  Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.

[55]  J. Kleinschmidt,et al.  Identification of a Heparin-Binding Motif on Adeno-Associated Virus Type 2 Capsids , 2003, Journal of Virology.

[56]  G. Deléage,et al.  Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2 , 1999, Nature Medicine.

[57]  James M. Wilson,et al.  Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors. , 2018, Human gene therapy methods.

[58]  N. Kanaan,et al.  Gene Therapy Models of Alzheimer's Disease and Other Dementias. , 2016, Methods in molecular biology.

[59]  J. Silverman,et al.  The Institutional Animal Care and Use Committee , 2015 .

[60]  K. Foust,et al.  Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes , 2009, Nature Biotechnology.