Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery

[1]  T. Kaisho,et al.  Effect of CpG Depletion of Vector Genome on CD8+ T Cell Responses in AAV Gene Therapy , 2021, Frontiers in Immunology.

[2]  J. F. Wright,et al.  Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes , 2021, Frontiers in Immunology.

[3]  H. Zetterberg,et al.  AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy , 2021, Gene Therapy.

[4]  J. Mendell,et al.  AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1 , 2021, Gene Therapy.

[5]  K. Flanigan,et al.  Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping , 2021, Human gene therapy.

[6]  M. Stavrou,et al.  Genetic mechanisms of peripheral nerve disease , 2020, Neuroscience Letters.

[7]  R. Tikkanen,et al.  Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[8]  J. Bennett,et al.  Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV , 2020, Molecular Therapy.

[9]  J. Mendell,et al.  Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy , 2020, JAMA neurology.

[10]  E. Smith,et al.  Intraarticular Adeno‐Associated Virus Serotype AAV‐PHP.S–Mediated Chemogenetic Targeting of Knee‐Innervating Dorsal Root Ganglion Neurons Alleviates Inflammatory Pain in Mice , 2020, Arthritis & rheumatology.

[11]  James M. Wilson,et al.  Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration , 2020, Molecular therapy. Methods & clinical development.

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

[13]  S. Gray,et al.  Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies , 2020, Brain Research.

[14]  I. Sargiannidou,et al.  Gene therapy approaches targeting Schwann cells for demyelinating neuropathies , 2019, Brain Research.

[15]  Shang Gao,et al.  Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS , 2019, Nature Medicine.

[16]  Caty Casas,et al.  Intrathecal AAVrh10 corrects Biochemical and Histological Hallmarks of Mucopolysaccharidosis VII Mice and Improves Behavior and Survival. , 2019, Human molecular genetics.

[17]  H. Zetterberg,et al.  Gene replacement therapy after neuropathy onset provides therapeutic benefit in a model of CMT1X. , 2019, Human molecular genetics.

[18]  C. Vite,et al.  Large animal models contribute to the development of therapies for central and peripheral nervous system dysfunction in patients with lysosomal storage diseases. , 2019, Human molecular genetics.

[19]  Anirudh Gupta,et al.  Charcot-Marie-Tooth: From Molecules to Therapy , 2019, International journal of molecular sciences.

[20]  Peter Bell,et al.  Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys. , 2019, Human gene therapy.

[21]  H. Zetterberg,et al.  Gene replacement therapy in a model of Charcot-Marie-Tooth 4C neuropathy , 2019, Brain : a journal of neurology.

[22]  G. Brenner,et al.  Schwannoma gene therapy by adeno-associated virus delivery of the pore-forming protein Gasdermin-D , 2019, Cancer Gene Therapy.

[23]  T. Conlon,et al.  Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration. , 2018, Journal of Huntington's disease.

[24]  James M. Wilson,et al.  Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques , 2018, Molecular therapy. Methods & clinical development.

[25]  C. Vite,et al.  AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease). , 2018, Human gene therapy.

[26]  D. Fuller,et al.  Sustained AAV9-mediated expression of a non-self protein in the CNS of non-human primates after immunomodulation , 2018, PloS one.

[27]  I. Sargiannidou,et al.  Intrathecal gene therapy in mouse models expressing CMT1X mutations , 2018, Human molecular genetics.

[28]  M. Yalvaç,et al.  AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model , 2018, Gene Therapy.

[29]  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.

[30]  D. Armao,et al.  Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy , 2015, Molecular therapy. Methods & clinical development.

[31]  K. Kleopa,et al.  Intrathecal Delivery of Viral Vectors for Gene Therapy. , 2018, Methods in molecular biology.

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

[33]  E. Bongarzone,et al.  Intrathecal administration of AAV/GALC vectors in 10–11‐day‐old twitcher mice improves survival and is enhanced by bone marrow transplant , 2016, Journal of neuroscience research.

[34]  W. Pardridge,et al.  CSF, blood-brain barrier, and brain drug delivery , 2016, Expert opinion on drug delivery.

[35]  G. Theophilidis,et al.  Intrathecal gene therapy rescues a model of demyelinating peripheral neuropathy , 2016, Proceedings of the National Academy of Sciences.

[36]  S. Scherer,et al.  Intraneural GJB1 gene delivery improves nerve pathology in a model of X‐linked Charcot–Marie–Tooth disease , 2015, Annals of neurology.

[37]  M. Barkats,et al.  Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice , 2015, Front. Mol. Neurosci..

[38]  W. Arnold,et al.  A large animal model of spinal muscular atrophy and correction of phenotype , 2015, Annals of neurology.

[39]  M. Chillón,et al.  AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy , 2014, Gene Therapy.

[40]  M. Chillón,et al.  Development of a rapid, robust, and universal picogreen-based method to titer adeno-associated vectors. , 2015, Human gene therapy methods.

[41]  B. Pukenas,et al.  Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna , 2014, Molecular therapy. Methods & clinical development.

[42]  L. Belur,et al.  Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse , 2014, Front. Neuroanat..

[43]  X. Navarro,et al.  Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy , 2014, Molecular therapy. Methods & clinical development.

[44]  James M. Wilson,et al.  Humoral Immune Response to AAV , 2013, Front. Immunol..

[45]  R. Samulski,et al.  Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates , 2012, Gene Therapy.

[46]  C. Mueller,et al.  Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

[47]  S. Ojeda,et al.  Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

[48]  R. Busuttil,et al.  RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy , 2010, The journal of gene medicine.

[49]  J. Fyfe,et al.  Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

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

[51]  P. Haydon,et al.  Cx29 and Cx32, two connexins expressed by myelinating glia, do not interact and are functionally distinct , 2008, Journal of Neuroscience Research.

[52]  D D Allen,et al.  The blood-brain barrier and brain drug delivery. , 2006, Journal of nanoscience and nanotechnology.

[53]  K. Fischbeck,et al.  Transgenic Expression of Human Connexin32 in Myelinating Schwann Cells Prevents Demyelination in Connexin32-Null Mice , 2005, The Journal of Neuroscience.

[54]  B. Byrne,et al.  Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield , 1999, Gene Therapy.