The Adeno-Associated virus - A safe and promising vehicle for liverspecific gene therapy of inherited and non-inherited disorders

Current Gene Therapy

Volumn 17, Issue 1, 2017, Pages 4-16

  Mak, Kai Y ^a   Rajapaksha, Indu G ^a   Angus, Peter W ^a,b   Herath, Chandana B ^a  

^a UNIVERSITY OF MELBOURNE   (Australia)

^b AUSTIN HEALTH   (Australia)

Author keywords

Adeno associated virus; Angiotensin converting enzyme 2; Gene therapy; Liver; Non inherited disorders; Renin angiotensin system; Viral vector

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA GALACTOSIDASE; ANGIOTENSIN CONVERTING ENZYME 2; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; CITRULLINEMIA; GLUCAN 1,4 ALPHA GLUCOSIDASE; HEPARIN SULFATE PROTEOGLYCAN; METHYLMALONYL COENZYME A MUTASE; MICRORNA; PHENYLALANINE 4 MONOOXYGENASE; PROTEOGLYCAN; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG;

ALPHA 1 ANTITRYPSIN DEFICIENCY; CAPSID ENGINEERING; CHOROIDEREMIA; CLINICAL TRIAL (TOPIC); COLOR BLINDNESS; DYSFERLINOPATHY; ENDOCYTOSIS; GENE EXPRESSION; GENE MUTATION; GENE OVEREXPRESSION; GENE SILENCING; GENE THERAPY; GENOME REPLICATION; HEMOPHILIA B; HOST CELL RECOGNITION; HUMAN; HUMAN ADENOVIRUS INFECTION; HURLER SYNDROME; IMMUNE RESPONSE; IN VIVO GENE TRANSFER; LIVER SPECIFIC GENE THERAPY; MOLECULAR DYNAMICS; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; NUCLEAR TRANSLOCATION; PARKINSON DISEASE; RECOGNITION; RETINITIS PIGMENTOSA; REVIEW; RHEUMATOID ARTHRITIS; SEX DIFFERENCE; THERAPEUTIC GENE TRANSFER; TISSUE SPECIFICITY; VIRAL GENE DELIVERY SYSTEM; VIRAL TROPISM; VIRION; VIRUS CAPSID; VIRUS ENTRY; VIRUS REPLICATION; VIRUS UNCOATING; VIRUS VIRULENCE; DEPENDOPARVOVIRUS; GENE VECTOR; GENETIC DISORDER; GENETICS; THERAPEUTIC USE; VIRUS GENOME;

DEPENDOVIRUS; GENETIC DISEASES, INBORN; GENETIC THERAPY; GENETIC VECTORS; GENOME, VIRAL; HUMANS;

EID: 85027299789     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/1566523217666170314141931     Document Type: Review

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