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1. Full Text Gene Therapy for Progressive Familial IntrahepaticCholestasis: Current Progress and Future Prospects
by Piter J Bosma and Marius Wits and Ronald P. J. Oude-Elferink
International journal of molecular sciences, ISSN 1661-6596, 12/2021, Volume 22, Issue 273, p. 273
PFIC | AAV | gene therapy
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2. Full Text AAV: An Overview of Unanswered Questions
by Berns, Kenneth I and Muzyczka, Nicholas
Human gene therapy, ISSN 1043-0342, 04/2017, Volume 28, Issue 4, pp. 38 - 313
Review Articles | AAV cell interactions | AAV persistence | AAV immunity | AAV toxicity | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Dependovirus - genetics | Genetic Vectors | Humans | Genetic Therapy - methods | Gene therapy | Index Medicus | Review
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3. Full Text Emerging Issues in AAV-Mediated In Vivo Gene Therapy
by Colella, Pasqualina and Ronzitti, Giuseppe and Mingozzi, Federico
Molecular therapy. Methods & clinical development, ISSN 2329-0501, 03/2018, Volume 8, Issue C, pp. 87 - 104
genotoxicity | inherited diseases | AAV | liver immunogenicity | AAV genome | gene therapy | persistence | AAV capsid | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Antigens | Neuromuscular diseases | Disease | Neurodegenerative diseases | Clinical trials | Viruses | Infections | Genomes | Drug development | Vectors (Biology) | Proteins | Plasmids | Blindness | Conflicts of interest | Biomarkers | Gene therapy | Drug dosages | Deoxyribonucleic acid--DNA | Methods | Expression vectors | Life Sciences | Human health and pathology
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4. Full Text Hypothalamic and other connections with dorsal CA2 area of the mouse hippocampus
by Cui, Zhenzhong and Gerfen, Charles R and Young, W. Scott
Journal of comparative neurology (1911), ISSN 0021-9967, 06/2013, Volume 521, Issue 8, pp. 1844 - 1866
median raphe nucleus | Green Beads | AAV‐GFP | vasopressin | entorhinal cortex | AAV‐tdTomato | hippocampal CA2 | septum | anterograde tracer | adeno‐associated virus | supramammillary nucleus | Fluorogold | cre recombinase | paraventricular nucleus | Adeno-associated virus | Supramammillary nucleus | Septum | AAV-GFP | Paraventricular nucleus | AAV-tdTomato | Cre recombinase | Vasopressin | Entorhinal cortex | Hippocampal CA2 | Anterograde tracer | Median raphe nucleus | Neurosciences | Neurosciences & Neurology | Life Sciences & Biomedicine | Zoology | Science & Technology | Gene Transfer Techniques | Green Fluorescent Proteins - metabolism | Brain - cytology | Stilbamidines - metabolism | Brain - anatomy & histology | Entorhinal Cortex - cytology | CA2 Region, Hippocampal - physiology | Hypothalamus - physiology | Male | Green Fluorescent Proteins - genetics | Functional Laterality | Neural Pathways - physiology | Indoles - metabolism | Animals | Neurons - physiology | Adenoviridae - genetics | Mice | Adenoviridae - metabolism | Plant Lectins - metabolism | Entorhinal Cortex - physiology | Plant Lectins - genetics | Index Medicus | Cortex (entorhinal) | adeno-associated virus
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5. Full Text Thermal Stability as a Determinant of AAV Serotype Identity
by Bennett, Antonette and Patel, Saajan and Mietzsch, Mario and Jose, Ariana and Lins-Austin, Bridget and Yu, Jennifer C and Bothner, Brian and McKenna, Robert and Agbandje-McKenna, Mavis
Molecular therapy. Methods & clinical development, ISSN 2329-0501, 09/2017, Volume 6, Issue C, pp. 171 - 182
AAV buffer formulations | AAV | differential scanning fluorimetry | AAV serotype identification | AAV vector buffers | parvovirus stability | adeno-associated virus | viral vectors | AAV capsid stability | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Peptides | Clinical trials | Amino acids | Cystic fibrosis | Identification | Genomes | Thermal stability | Capsids | Rheumatoid arthritis | Hemophilia | Genetic engineering | Neuronal ceroid lipofuscinosis | Gene therapy | Heart diseases | Methods
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6. Full Text Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome
by Aronson, S.J and Veron, P and Collaud, F and Hubert, A and Delahais, V and Honnet, G and Knegt, Robert and Junge, N and Baumann, Ulrich and Di Giorgio, A and D'Antiga, L and Ginocchio, V.M and Brunetti-Pierri, Nicola and Labrune, P and Beuers, Ulrich and Bosma, Piter and Mingozzi, F
Human gene therapy, ISSN 1043-0342, 10/2019, Volume 30, Issue 10, pp. 1297 - 1305
AAV gene therapy | Crigler–Najjar syndrome | UGT1A1 | pre-existing immunity | unconjugated hyperbilirubinemia | anti-AAV neutralizing antibodies
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7. Full Text Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression
by Martinez-Navio, José M and Fuchs, Sebastian P and Pantry, Shara N and Lauer, William A and Duggan, Natasha N and Keele, Brandon F and Rakasz, Eva G and Gao, Guangping and Lifson, Jeffrey D and Desrosiers, Ronald C
Immunity (Cambridge, Mass.), ISSN 1074-7613, 03/2019, Volume 50, Issue 3, pp. 567 - 575.e5
AAV vector | anti-drug antibodies | functional cure | HIV broadly neutralizing antibodies | rhesus macaques | immunotherapy | SHIV infection | viral reservoir | gene therapy | adeno-associated virus | Life Sciences & Biomedicine | Immunology | Science & Technology | Cell Line | Humans | Broadly Neutralizing Antibodies | Dependovirus - immunology | Macaca mulatta | Antibodies, Monoclonal, Humanized | HIV Infections - immunology | Antibodies, Neutralizing - immunology | HIV-1 - immunology | Animals | HIV Antibodies - immunology | HEK293 Cells | Viremia - immunology | Antibodies, Monoclonal - immunology | Virus diseases | Prevention | Medical colleges | Biological products | Genetic vectors | Primates | Monoclonal antibodies | Biological products industry | HIV (Viruses) | Health aspects | HIV infection | Plasma | Medical research | Drug delivery systems | Immunoglobulins | Viremia | Viruses | Infections | Monkeys | Monkeys & apes | Acquired immune deficiency syndrome--AIDS | Coding | Human immunodeficiency virus--HIV | Binding sites | Cancer | antibody | AAV delivery | AAV
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8. Full Text Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors
by Kruzik, Anita and Fetahagic, Damir and Hartlieb, Bettina and Dorn, Sebastian and Koppensteiner, Herwig and Horling, Frank M and Scheiflinger, Friedrich and Reipert, Birgit M and de la Rosa, Maurus
Molecular therapy. Methods & clinical development, ISSN 2329-0501, 09/2019, Volume 14, pp. 126 - 133
clinical assays | anti-AAV prevalence | anti-AAV antibody response | AAV | gene therapy | adeno-associated virus | international cohorts | anti-AAV T cell response | Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Interleukin 2 | Lymphocytes | Immunoglobulin G | γ-Interferon | Standardization | Hemophilia | Clinical trials | Serotypes | Employees | Lymphocytes T | Gene therapy | Enzyme-linked immunosorbent assay
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9. Full Text Gene delivery to neurons in the auditory brainstem of barn owls using standard recombinant adeno-associated virus vectors
by Thiele, Nadine and Hildebrandt, K. Jannis and Köppl, Christine
Current Research in Neurobiology, ISSN 2665-945X, 12/2020, Volume 1
Avian | Optogenetics | AAV | Bird
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10. Full Text Lung biopsy in the diagnosis of pediatric ANCA‐associated vasculitis
by Sayad, Edouard and Vogel, Tiphanie P and Cortes‐Santiago, Nahir and Patel, Kalyani R and McNeill, David M and Spielberg, David and Silva‐Carmona, Manuel
Pediatric pulmonology, ISSN 8755-6863, 01/2021, Volume 56, Issue 1, pp. 145 - 152
microscopic polyangiitis (MPA) | lung histopathology | pediatric vasculitis | lung biopsy | pulmonary hemorrhage | granulomatosis with polyangiitis (GPA) | anti‐neutrophil cytoplasmic antibody‐associated vasculitis (AAV) | pediatric AAV | anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) | Respiratory System | Pediatrics | Life Sciences & Biomedicine | Science & Technology | Medical research | Lung diseases | Antibodies | Viral antibodies | Vasculitis | Lungs | Biopsy | Medicine, Experimental | Diagnosis | Children | Health aspects | Histochemistry | Serology | Histopathology | Index Medicus
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