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Blood, ISSN 1528-0020, 2015, Volume 126, Issue 15, pp. 1777 - 1784
Journal Article
Human gene therapy, ISSN 1557-7422, 2008, Volume 19, Issue 10, pp. 979 - 990
Journal Article
Science (American Association for the Advancement of Science), ISSN 1095-9203, 2010, Volume 329, Issue 5990, pp. 413 - 417
Journal Article
Nature genetics, ISSN 1546-1718, 2014, Volume 46, Issue 4, pp. 352 - 356
Journal Article
Molecular Therapy, ISSN 1525-0016, 2008, Volume 16, Issue 9, pp. 1630 - 1636
Journal Article
Molecular therapy, ISSN 1525-0016, 2017, Volume 25, Issue 5, pp. 1168 - 1186
... and pre-clinical settings for the potential treatment of genetic diseases, cancer, and infectious diseases. The majority of these applications are attributed... 
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 | Original
Journal Article
Nature communications, ISSN 2041-1723, 2018, Volume 9, Issue 1, pp. 1855 - 8
LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that... 
CELLS | SPECIFICITIES | ENDONUCLEASE | EFFICACY | INJECTION | MULTIDISCIPLINARY SCIENCES | CPF1 | CRISPR-CAS | EXPRESSION | ENDOTHELIAL GROWTH-FACTOR | NUCLEASES | Dependovirus - genetics | Endonucleases - genetics | Genetic Vectors - administration & dosage | Intravitreal Injections | RNA, Guide - genetics | Humans | Choroidal Neovascularization - therapy | Choroidal Neovascularization - etiology | Male | Vascular Endothelial Growth Factor A - genetics | Retinal Pigment Epithelium - pathology | Choroidal Neovascularization - pathology | Endonucleases - administration & dosage | Retinal Pigment Epithelium - blood supply | Clostridiales - genetics | Retinal Pigment Epithelium - radiation effects | Clustered Regularly Interspaced Short Palindromic Repeats - genetics | Disease Models, Animal | Lasers - adverse effects | Macular Degeneration - etiology | Cell Line | Specific Pathogen-Free Organisms | Hypoxia-Inducible Factor 1, alpha Subunit - genetics | Mice, Inbred C57BL | Bacterial Proteins - genetics | Treatment Outcome | Bacterial Proteins - administration & dosage | Genetic Vectors - genetics | Animals | Macular Degeneration - genetics | Macular Degeneration - therapy | Gene Editing - methods | Mice | Choroidal Neovascularization - genetics | Genetic Therapy - methods | Macular Degeneration - pathology | CRISPR | Therapeutic applications | Medical treatment | Viruses | Retina | Genomes | Ribonucleic acid--RNA | Epithelium | Age related diseases | Retinal pigment epithelium | Vascularization | Macular degeneration | Angiogenesis | Gene disruption | Hypoxia | Endonuclease | Vascular endothelial growth factor | Genetic modification | Age
Journal Article
Journal Article