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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
Journal Article
Current Gene Therapy, ISSN 1566-5232, 06/2005, Volume 5, Issue 3, pp. 285 - 297
Journal Article
Journal of Comparative Neurology, ISSN 0021-9967, 06/2013, Volume 521, Issue 8, pp. 1844 - 1866
Journal Article
Transfusion and Apheresis Science, ISSN 1473-0502, 08/2019
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 09/2017, Volume 6, Issue C, pp. 171 - 182
Journal Article
Molecular Therapy - Methods & Clinical Development, ISSN 2329-0501, 03/2018, Volume 8, Issue C, pp. 87 - 104
Journal Article
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
Journal Article
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
Journal Article
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
Journal Article
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
Journal Article
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
Journal Article
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
Journal Article
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
Journal Article
Journal of Cellular and Molecular Medicine, ISSN 1582-1838, 11/2018, Volume 22, Issue 11, pp. 5583 - 5595
Journal Article