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Journal of Clinical Investigation, ISSN 0021-9738, 05/2008, Volume 118, Issue 5, pp. 1955 - 1964
Journal Article
Applied and Environmental Microbiology, ISSN 0099-2240, 2017, Volume 83, Issue 5, p. E03005
Journal Article
Human Gene Therapy, ISSN 1043-0342, 10/2008, Volume 19, Issue 10, pp. 979 - 990
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 02/2014, Volume 6, Issue 2, pp. 194 - 211
Retinal gene therapy with adeno‐associated viral ( AAV ) vectors is safe and effective in humans. However, AAV 's limited cargo capacity prevents its... 
ABCA | gene therapy | MYO | retina | AAV | Retina | MYO7A | Gene therapy | ABCA4 | MEDICINE, RESEARCH & EXPERIMENTAL | ROD OUTER SEGMENTS | PACKAGING CAPACITY | USHER-SYNDROME 1B | MACULAR DEGENERATION | MYOSIN VIIA | ADENOASSOCIATED VIRAL VECTORS | RIM PROTEIN | IN-VIVO | LEBERS CONGENITAL AMAUROSIS | VIRUS VECTORS | Retinal Pigment Epithelium - metabolism | Usher Syndromes - therapy | Retina - metabolism | Genetic Vectors - administration & dosage | Humans | Rhodopsin - metabolism | Usher Syndromes - genetics | HEK293 Cells | ATP-Binding Cassette Transporters - metabolism | Myosins - metabolism | Disease Models, Animal | Gene Transfer Techniques | Transduction, Genetic | Dependovirus - metabolism | Mice, Inbred C57BL | Genetic Vectors - metabolism | Injections | Phenotype | Animals | Macular Degeneration - congenital | Macular Degeneration - genetics | Macular Degeneration - therapy | Mice | Photoreceptor Cells, Vertebrate - metabolism | Lipofuscin - metabolism | Melanosomes - metabolism | Sus scrofa | Trans-Splicing - genetics | Genetic research | Swine | Analysis | Genes | Genomics | Phenotypes | Animal models | Disease | Splicing | Homologous recombination | Infections | Genomes | Research | Gene expression | Phosphatase | Experiments | Vectors (Biology) | Capsids | Hogs | Photoreceptors | Mutation | Expression vectors | Index Medicus
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1168 - 1186
CRISPR-associated protein 9 (Cas9)-mediated genome editing provides a promising cure for HIV-1/AIDS; however, gene delivery efficiency in vivo remains an... 
genome editing | humanized BLT mice | animal models | viral latency | HIV | AAV | Tg26 mice | gene therapy | Cas9 | gRNA | EFFICIENT DELIVERY | MEDICINE, RESEARCH & EXPERIMENTAL | GENE EDITING TECHNOLOGIES | PACKAGING CAPACITY | LENTIVIRAL VECTORS | ADENOASSOCIATED VIRAL VECTORS | STAPHYLOCOCCUS-AUREUS CAS9 | CRISPR-CAS9 NUCLEASES | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | CRISPR/CAS9 SYSTEM | GENETICS & HEREDITY | MODULATES PROLIFERATION | NEURAL PRECURSOR CELLS | Oligonucleotides - genetics | Dependovirus - genetics | Endonucleases - genetics | Staphylococcus aureus - enzymology | RNA, Guide - genetics | RNA, Guide - metabolism | Humans | HIV Long Terminal Repeat | pol Gene Products, Human Immunodeficiency Virus - metabolism | Endonucleases - metabolism | gag Gene Products, Human Immunodeficiency Virus - metabolism | HIV Infections - pathology | Proviruses - genetics | Base Sequence | Clustered Regularly Interspaced Short Palindromic Repeats | gag Gene Products, Human Immunodeficiency Virus - genetics | pol Gene Products, Human Immunodeficiency Virus - genetics | Disease Models, Animal | Genome, Viral | HIV-1 - metabolism | Dependovirus - metabolism | Genetic Vectors - chemistry | HIV Infections - virology | Bacterial Proteins - genetics | Genetic Vectors - metabolism | Mice, Transgenic | HIV-1 - genetics | Proviruses - metabolism | Staphylococcus aureus - chemistry | Oligonucleotides - metabolism | Animals | CRISPR-Cas Systems | HIV Infections - therapy | Bacterial Proteins - metabolism | Gene Editing - methods | Mice | Genetic Therapy - methods | Neuroimaging | Animal models | Bioluminescence | Intravenous administration | Genes | Liver | Clinical trials | Chronic infection | Infections | Genomes | Thymus | Acquired immune deficiency syndrome--AIDS | Efficiency | Transgenic animals | Human immunodeficiency virus--HIV | Bone marrow | Colon | Deoxyribonucleic acid--DNA | Spleen | CRISPR | Gene transfer | Advisors | Transgenic mice | Ribonucleic acid--RNA | Progenitor cells | Polymerase chain reaction | Studies | Genotyping | Neural stem cells | Index Medicus | Original
Journal Article