المؤلفون: Ayumu Konno, Yoichiro Shinohara, Hirokazu Hirai

المصدر: International Journal of Molecular Sciences, Vol 25, Iss 13, p 7205 (2024)

مصطلحات موضوعية: adeno-associated virus, AAV, AAV-PHP.B, Spinocerebellar ataxia type 3, SCA3, Machado–Joseph disease, Biology (General), QH301-705.5, Chemistry, QD1-999

Relation: https://www.mdpi.com/1422-0067/25/13/7205; https://doaj.org/toc/1661-6596; https://doaj.org/toc/1422-0067; https://doaj.org/article/6da3aff01b2e4481bb50316fbac6750e

الاتاحة: https://doi.org/10.3390/ijms25137205
https://doaj.org/article/6da3aff01b2e4481bb50316fbac6750e

المؤلفون: Liguore, William, Domire, Jacqueline, Button, Dana, Wang, Yun, Dufour, Brett, Srinivasan, Sathya, McBride, Jodi

المصدر: Molecular Therapy. 27(11)

مصطلحات موضوعية: AAV-PHP.B, AAV9, CNS, adeno-associated virus, brain, gene therapy, gene transfer, mouse, rhesus macaque, viral transduction, Animals, Brain, Central Nervous System, Dependovirus, Disease Models, Animal, Fluorescent Antibody Technique, Gene Expression, Gene Transfer Techniques, Genes, Reporter, Genetic Therapy, Genetic Vectors, Humans, Macaca mulatta, Mice, Neurons, Spinal Cord, Tissue Distribution, Transduction, Genetic, Transgenes

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/0tn24818
https://escholarship.org/content/qt0tn24818/qt0tn24818.pdf

المؤلفون: Chiaki Hoshino (10146968), Ayumu Konno (10146971), Nobutake Hosoi (10146974), Ryosuke Kaneko (2795341), Ryo Mukai (6204680), Junichi Nakai (113324), Hirokazu Hirai (10146977)

مصطلحات موضوعية: Biochemistry, Medicine, Cell Biology, Genetics, Molecular Biology, Neuroscience, Physiology, Pharmacology, Developmental Biology, Mental Health, Infectious Diseases, Biological Sciences not elsewhere classified, Interneuron, Inhibitory neuron, Chandelier cell, GAD65, Dlx, GABA, Promoter, AAV, AAV-PHP.B

Relation: https://figshare.com/articles/journal_contribution/Additional_file_1_of_GABAergic_neuron-specific_whole-brain_transduction_by_AAV-PHP_B_incorporated_with_a_new_GAD65_promoter/14037551

الاتاحة: https://doi.org/10.6084/m9.figshare.14037551.v1

المؤلفون: Lim, Jeong-A, Yi, Haiqing, Gao, Fengqin, Raben, Nina, Kishnani, Priya S, Sun, Baodong

مصطلحات موضوعية: AAV-PHP.B vector, CNS, Pompe disease, acid alpha-glucosidase deficiency, gene therapy, glycogen storage, neurological deficits

وصف الملف: application/pdf

Relation: Molecular therapy. Methods & clinical development; S2329-0501(19)30009-9; https://hdl.handle.net/10161/27503

الاتاحة: https://hdl.handle.net/10161/27503

المؤلفون: Yang Gao, Liyi Geng, Vicky Chen, Stephen Brimijoin

المصدر: Molecules; Volume 22; Issue 7; Pages: 1145

مصطلحات موضوعية: butyrylcholinesterase, adeno-associated virus (AAV)-8, AAV-9, AAV-PHP.B, enzyme expression, ghrelin

جغرافية الموضوع: agris

وصف الملف: application/pdf

Relation: Medicinal Chemistry; https://dx.doi.org/10.3390/molecules22071145

الاتاحة: https://doi.org/10.3390/molecules22071145

المؤلفون: Batista, Ana Rita, King, Oliver D., Reardon, Christopher P., Davis, Crystal, Shankaracharya, FNU, Philip, Vivek, Gray-Edwards, Heather L, Aronin, Neil, Lutz, Cathleen, Landers, John E, Sena-Esteves, Miguel

المساهمون: RNA Therapeutics Institute, Department of Radiology, Horae Gene Therapy Center, Department of Neurology

المصدر: Human gene therapy ; 31 ; 1-2 ; 90-102

مصطلحات موضوعية: Animals, Antigens, Ly, Blood-Brain Barrier, Central Nervous System, Dependovirus, Endothelium, Vascular, Female, Gene Expression, Gene Transfer Techniques, Genes, Reporter, Genetic Vectors, Genotype, Male, Membrane Proteins, Mice, Transgenic, Quantitative Trait Loci, Species Specificity, Transduction, Genetic, AAV-PHP.B, CNS transduction, Ly6a, blood–brain barrier, mouse genetics, Genetics and Genomics, Nervous System

Relation: View article in PubMed; https://doi.org/10.1089/hum.2019.186; Batista AR, King OD, Reardon CP, Davis C, Shankaracharya, Philip V, Gray-Edwards H, Aronin N, Lutz C, Landers J, Sena-Esteves M. Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B. Hum Gene Ther. 2020 Jan;31(1-2):90-102. doi:10.1089/hum.2019.186. Epub 2019 Dec 13. PMID: 31696742.; http://hdl.handle.net/20.500.14038/29846; https://escholarship.umassmed.edu/faculty_pubs/2052; faculty_pubs/2052

الاتاحة: https://doi.org/10.1089/hum.2019.186
https://hdl.handle.net/20.500.14038/29846
https://escholarship.umassmed.edu/faculty_pubs/2052

المؤلفون: Nina Raben, Fengqin Gao, Haiqing Yi, Baodong Sun, Priya S. Kishnani, Jeong-A Lim

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 233-245 (2019)
Molecular Therapy. Methods & Clinical Development

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, lcsh:QH426-470, Genetic enhancement, glycogen storage, Article, acid alpha-glucosidase deficiency, Virus, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, AAV-PHP.B vector, Internal medicine, Glycogen storage disease type II, Genetics, medicine, Vector (molecular biology), lcsh:QH573-671, neurological deficits, Molecular Biology, Glycogen, lcsh:Cytology, business.industry, Pompe disease, Skeletal muscle, Enzyme replacement therapy, medicine.disease, Spinal cord, gene therapy, lcsh:Genetics, 030104 developmental biology, medicine.anatomical_structure, Endocrinology, chemistry, 030220 oncology & carcinogenesis, Molecular Medicine, CNS, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c456e91a24b288f5fdaf414228066cc8
https://doi.org/10.1016/j.omtm.2019.01.006

المؤلفون: Santoso, Putri Teesa Radhiyanti

مصطلحات موضوعية: NSE promoter, Adeno-associated virus, CaMKII promoter, AAV-PHP.B, Synapsin I promoter, neuron-specific promotor

URL الوصول: https://explore.openaire.eu/search/publication?articleId=jairo_______::9c3f8ba7db59c3da8cca65aec80e9c29
http://hdl.handle.net/10087/13541

المؤلفون: Hirokazu Hirai, Ayumu Konno, Ryosuke Kaneko, Nobutake Hosoi, Ryo Mukai, Junichi Nakai, Chiaki Hoshino

المصدر: Molecular Brain
Molecular Brain, Vol 14, Iss 1, Pp 1-18 (2021)

مصطلحات موضوعية: 0301 basic medicine, Glutamate decarboxylase, AAV-PHP.B, lcsh:RC346-429, GABA, 0302 clinical medicine, Transduction, Genetic, Chandelier cell, GABAergic Neurons, Promoter Regions, Genetic, Neurons, biology, Glutamate Decarboxylase, Motor Cortex, Brain, AAV, Dependovirus, Cell biology, medicine.anatomical_structure, Parvalbumins, Injections, Intravenous, GABAergic, Plasmids, Interneuron, Dlx, Green Fluorescent Proteins, Inhibitory postsynaptic potential, 03 medical and health sciences, Cellular and Molecular Neuroscience, Interneurons, medicine, Animals, Humans, Enhancer, Molecular Biology, lcsh:Neurology. Diseases of the nervous system, Research, Promoter, Inhibitory neuron, Axon initial segment, Mice, Inbred C57BL, 030104 developmental biology, HEK293 Cells, nervous system, GAD65, biology.protein, Calcium, 030217 neurology & neurosurgery, Parvalbumin

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cded78f5f1b9adc582afad15e712b965
https://pubmed.ncbi.nlm.nih.gov/33588899

المؤلفون: Santoso, Putri Teesa Radhiyanti, サントソ, プトリ テッサ ラディヤンティ

مصطلحات موضوعية: Adeno-associated virus, AAV-PHP.B, neuron-specific promotor, CaMKII promoter, NSE promoter, Synapsin I promoter

Relation: http://hdl.handle.net/10087/13541

الاتاحة: http://hdl.handle.net/10087/13541

المؤلفون: Martino, Richard, Alexander

المساهمون: Wilson, James, M

مصطلحات موضوعية: Life Sciences, AAV-PHP.B, Adeno-Associated Virus (AAV), Blood-Brain Barrier, Drug Delivery, Gene Therapy, Ly6a

وصف الملف: application/pdf

Relation: https://repository.upenn.edu/handle/20.500.14332/59702

الاتاحة: https://repository.upenn.edu/handle/20.500.14332/59702
https://hdl.handle.net/20.500.14332/59702