Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery
暂无分享,去创建一个
I. Sargiannidou | K. Kleopa | A. Kagiava | J. Richter | C. Tryfonos | C. Christodoulou | A. Bosch | M. Leal-Julià
[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.