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1. Full Text Design of AAV vectors for delivery of large or multiple transgenes
by Patel, Aman and Zhao, Junling and Duan, Dongsheng and Lai, Yi
Methods in Molecular Biology, ISSN 1064-3745, 2019, Volume 1950, pp. 19 - 33
Adeno-associated virus (AAV)-mediated gene therapy has evolved from bench to bedside, and now is the therapy of choice for certain inherited diseases. However,... 
Fragmented AAV gene delivery | Large gene expression cassette | Triple vectors | Trans-splicing AAV vectors | Overlapping AAV vectors | AAV | Dual vectors | Split AAV vectors | Multiple gene expression cassette | Oversized AAV
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2. New recombinant serotypes of AAV vectors
by Gao, Guangping and Vandenberghe, Luk H and Wilson, James M
Current Gene Therapy, ISSN 1566-5232, 06/2005, Volume 5, Issue 3, pp. 285 - 297
AAV based vectors can achieve stable gene transfer with minimal vector related toxicities. AAV serotype 2 (AAV2) is the first AAV that was vectored for gene... 
tissue tropism | AAV serotypes | animal models | AAV evolution | gene transfer | PARTICLES | AAV clades | ERYTHROPOIETIN GENE-THERAPY | TRANSDUCTION | VIRAL VECTORS | CLONING | IN-VIVO | GENETICS & HEREDITY | ADENOASSOCIATED VIRUS VECTORS | ADENOVIRUS-ASSOCIATED VIRUSES | AAV vectors | AUTOIMMUNE ANEMIA | HUMAN PARVOVIRUS | AAV sero-epidemiology | Dependovirus - genetics | Gene Transfer Techniques | Genetic Therapy | Genome, Viral | Animals | Recombination, Genetic | Humans | Dependovirus - classification | Phylogeny | Genetic Vectors
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3. 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, ISSN 0021-9967, 06/2013, Volume 521, Issue 8, pp. 1844 - 1866
The CA2 area is an important, although relatively unexplored, component of the hippocampus. We used various tracers to provide a comprehensive analysis of CA2... 
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 | VASOPRESSIN 1B RECEPTOR | AGGRESSIVE-BEHAVIOR | PROJECTION PATTERNS | adeno-associated virus | THETA-RHYTHM | NEUROSCIENCES | SEPTAL AREA | ZOOLOGY | PYRAMIDAL CELLS | ANTEROGRADE TRANSPORT | 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 | Cortex (entorhinal)
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4. Full Text Gene therapy for hemophilia: Progress to date and challenges moving forward
by Gollomp, Kandace L and Doshi, Bhavya S and Arruda, Valder R
Transfusion and Apheresis Science, ISSN 1473-0502, 08/2019
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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
Currently, there are over 150 ongoing clinical trials utilizing adeno-associated viruses (AAVs) to target various genetic diseases, including hemophilia (AAV2... 
AAV buffer formulations | AAV | differential scanning fluorimetry | AAV serotype identification | AAV vector buffers | parvovirus stability | adeno-associated virus | viral vectors | AAV capsid stability | ADENOASSOCIATED VIRUS TYPE-2 | MEDICINE, RESEARCH & EXPERIMENTAL | CAPSID PROTEIN | PURIFICATION | GENE-THERAPY | RAAV VECTORS | HIGH-TITER | IDENTIFICATION | EXPRESSION | TISSUE-TROPISM | DELIVERY
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6. 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
In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors have been used for gene transfer has steadily increased. The... 
genotoxicity | inherited diseases | AAV | liver immunogenicity | gene therapy | persistence | AAV genome | AAV capsid | MEDICINE, RESEARCH & EXPERIMENTAL | NONHUMAN PRIMATE MODEL | T-CELL RESPONSES | LONG-TERM SAFETY | FACTOR-IX | MOUSE MODEL | FACTOR-VIII | ADENOASSOCIATED VIRUS VECTOR | ACID ALPHA-GLUCOSIDASE | ADAPTIVE IMMUNE-RESPONSES | HEMOPHILIA-B DOGS | Life Sciences | Human health and pathology
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7. Full Text AAV vectors for efficient gene delivery to rodent hearts
by Lopez-Gordo, Estrella and Kohlbrenner, Erik and Katz, Michael G and Weber, Thomas
Methods in Molecular Biology, ISSN 1064-3745, 2019, Volume 1950, pp. 311 - 332
Currently, gene therapy is one of the most promising fields in biomedicine, with great therapeutic potential for an array of inherited and acquired diseases.... 
Adeno-associated virus | Mouse | Rat | AAV delivery | AAV production | AAV characterization | AAV9
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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
Preexisting immunity against adeno-associated virus (AAV) is a major challenge facing AAV gene therapy, resulting in the exclusion of patients from clinical... 
clinical assays | anti-AAV prevalence | anti-AAV antibody response | AAV | gene therapy | adeno-associated virus | international cohorts | anti-AAV T cell response | MEDICINE, RESEARCH & EXPERIMENTAL | T-CELL RESPONSES | NEUTRALIZING ANTIBODIES | RECOMBINANT ADENOASSOCIATED VIRUS | MEMORY | LIVER | TRANSDUCTION | VECTORS
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9. Full Text Seeing the Light after 25 Years of Retinal Gene Therapy
by Trapani, Ivana and Auricchio, Alberto
Trends in Molecular Medicine, ISSN 1471-4914, 08/2018, Volume 24, Issue 8, pp. 669 - 681
The retina has been at the forefront of translational gene therapy. Proof-of-concept that gene therapy could restore vision in a large animal led to the... 
retina | gene therapy | AAV
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10. Full Text AAV serotype 2/1-mediated gene delivery of anti-inflammatory interleukin-10 enhances neurogenesis and cognitive function in APPPS1 mice
by Kiyota, T and Ingraham, K.L and Swan, R.J and Jacobsen, M.T and Andrews, S.J and Ikezu, T
Gene Therapy, ISSN 0969-7128, 07/2012, Volume 19, Issue 7, pp. 724 - 733
neurogenesis | neuroimmunology | cytokines | Alzheimer's disease | AAV | β-amyloid
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11. Full Text Reduced CD5 + CD24 hi CD38 hi and interleukin-10 + regulatory B cells in active anti-neutrophil cytoplasmic autoantibody-associated vasculitis permit increased circulating autoantibodies
by Aybar, L.T and Mcgregor, J.G and Hogan, S.L and Hu, Y and Mendoza, C.E and Brant, E.J and Poulton, C.J and Henderson, C.D and Falk, R.J and Bunch, D.O
Clinical and Experimental Immunology, ISSN 0009-9104, 05/2015, Volume 180, Issue 2, pp. 178 - 188
Regulatory B cells | Interleukin-10 | ANCA | CD5 | AAV
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12. Full Text Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
by Perocheau, Dany P and Cunningham, Sharon C and Lee, Juhee and Antinao Diaz, Juan and Waddington, Simon N and Gilmour, Kimberly and Eaglestone, Simon and Lisowski, Leszek and Thrasher, Adrian J and Alexander, Ian E and Gissen, Paul and Baruteau, Julien
Human Gene Therapy, ISSN 1043-0342, 01/2019, Volume 30, Issue 1, pp. 79 - 87
Recombinant adeno-associated virus (rAAV) vectors are a promising platform for in vivo gene therapy. The presence of neutralizing antibodies (Nab) against AAV... 
Research Articles | Adeno-associated virus | Neutralizing antibodies | AAV | Liver | AAV-LK03 | Gene therapy | Seroprevalence | MEDICINE, RESEARCH & EXPERIMENTAL | AAV VECTORS | liver | seroprevalence | gene therapy | neutralizing antibodies | adeno-associated virus | OPEN-LABEL | HEMOPHILIA | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IMMUNE-RESPONSES | LONG-TERM SAFETY | FACTOR-IX | GENETICS & HEREDITY | GENE-THERAPY | PHASE-I | LIVER TRANSDUCTION
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13. Full Text Molecular Detection of Adeno-Associated Virus DNA in Cases of Spontaneous and Therapeutic Abortion
by Sayyadi-Dehno, Zahra and Seyed Khorrami, Seyed Mahmood and Ghavami, Nastaran and Ghotbi-Zadeh, Fahimeh and Khushideh, Maryam and Hosseini, Mostafa and Malekshahi, Somayeh Shatizadeh and Shafiei-Jandaghi, Nazanin-Zahra
Fetal and Pediatric Pathology, ISSN 1551-3815, 05/2019, Volume 38, Issue 3, pp. 206 - 214
Background: Adeno-associated viruses (AAVs) have been found in human blood cells, cervical biopsies, and epithelial cell brushings, endometrium, and abortion... 
therapeutic abortion | spontaneous abortion | AAV
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14. Full Text Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency
by Matsumura, Yuki and Stiles, Katie M and Reid, Jasmine and Frenk, Esther Z and Cronin, Samantha and Pagovich, Odelya E and Crystal, Ronald G
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 12/2019, Volume 15, pp. 72 - 82
Aldehyde dehydrogenase 2 (ALDH2) deficiency causes “Asian flush syndrome,” presenting as alcohol-induced facial flushing, tachycardia, nausea, and headaches.... 
gene therapy | acetaldehyde | ALDH2 | AAV
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15. Full Text Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina
by Trapani, I
GENES, ISSN 2073-4425, 04/2019, Volume 10, Issue 4, p. 287
Gene therapy using adeno-associated viral (AAV) vectors currently represents the most promising approach for the treatment of many inherited retinal diseases... 
SYSTEM | AAV VECTORS | VIRUS | dual AAV | PACKAGING CAPACITY | RECONSTRUCTION | gene therapy | TRANSDUCTION | THERAPY | retina | AAV | MOUSE MODEL | GENETICS & HEREDITY | ANIMAL-MODELS | EXPRESSION
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16. Full Text Intramuscular delivery of HGF-expressing recombinant AAV improves muscle integrity and alleviates neurological symptoms in the nerve crush and SOD1-G93A transgenic mouse models
by Lee, Sang Hwan and Lee, Nayeon and Lee, Junghun and Kim, Jin Hong and Kim, Sunyoung and Kim, Subin and Choi, Wooshik and Yu, Seung Shin
Biochemical and Biophysical Research Communications, ISSN 0006-291X, 09/2019, Volume 517, Issue 3, pp. 452 - 457
Hepatocyte growth factor (HGF) is a versatile neurotrophic factor that mediates a variety of cellular activities. In this study, we investigated the effects of... 
ALS | IM injection | Sciatic nerve crush | AAV | HGF
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17. Full Text Gene Transfer of ZMapp Antibodies Mediated by Recombinant Adeno-Associated Virus Protects Against Ebola Infections
by Robert, Marc-André and Nassoury, Nasha and Chahal, Parminder S and Venne, Marie-Hélène and Racine, Trina and Qiu, Xiangguo and Kobinger, Gary and Kamen, Amine and Gilbert, Rénald and Gaillet, Bruno
Human Gene Therapy, ISSN 1043-0342, 04/2018, Volume 29, Issue 4, pp. 452 - 466
Vectored delivery of the ZMapp antibody cocktail (c2G4, c4G7, and c13C6) by using recombinant adeno-associated viruses (rAAVs) could be useful for preventive... 
Research Articles | Ebola virus | mouse | antibody gene transfer | preventive treatment | AAV | ZMapp | AAV manufacturing | humoral response | TRANSIENT TRANSFECTION | EFFICIENT TRANSDUCTION | MEDICINE, RESEARCH & EXPERIMENTAL | THERAPEUTIC LEVELS | SPINAL-CORD | ADENOVIRAL VECTORS | TROPONIN-I GENE | LEBER CONGENITAL AMAUROSIS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | SERUM-FREE PRODUCTION | IMMUNE-RESPONSES | GENETICS & HEREDITY | AAV SEROTYPES | Immunization | Immunoglobulins | Intravenous administration | Effectiveness | Gene transfer | Antibodies | Viruses | Genomes | Infections | Transfection | Bioreactors | Mice | Survivability | Recombinant
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18. Full Text Notch pathway activation enhances cardiosphere in vitro expansion
by Secco, Ilaria and Barile, Lucio and Torrini, Consuelo and Zentilin, Lorena and Vassalli, Giuseppe and Giacca, Mauro and Collesi, Chiara
Journal of Cellular and Molecular Medicine, ISSN 1582-1838, 11/2018, Volume 22, Issue 11, pp. 5583 - 5595
Cardiospheres (CSps) are self‐assembling clusters of a heterogeneous population of poorly differentiated cells outgrowing from in vitro cultured cardiac... 
Adeno‐associated virus (AAV) | Cardiac Progenitors | Notch | Micro‐RNAs | Adeno-associated virus (AAV) | Micro-RNAs | MEDICINE, RESEARCH & EXPERIMENTAL | STEM-CELLS | REGENERATION | CONSENSUS | PROLIFERATION | FATE | CELL BIOLOGY | HEART | INHIBITION | GROWTH-FACTORS | DIFFERENTIATION | LIGAND | Heart | MicroRNA | Gene expression | Health aspects | Explants | Gene transfer | Viruses | Cardiomyocytes | Event-related potentials | Activation | Kinases | Chromosome 3 | Signal transduction | Toxicology | Jagged1 protein | Differentiation | Original
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