Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Human Molecular Genetics

Volumn 24, Issue 23, 2015, Pages 6811-6825

  Trapani, Ivana ^a   Toriello, Elisabetta ^a   De Simone, Sonia ^a   Colella, Pasqualina ^a   Iodice, Carolina ^a   Polishchuk, Elena V ^a   Sommella, Andrea ^a   Colecchi, Linda ^a   Rossi, Settimio ^b   Simonelli, Francesca ^b   Giunti, Massimo ^c   Bacci, Maria L ^c   Polishchuk, Roman S ^a   Auricchio, Alberto ^a,d  

^a TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^b SECOND UNIVERSITY OF NAPLES   (Italy)

^c UNIVERSITY OF BOLOGNA   (Italy)

^d UNIVERSITY OF NAPLES FEDERICO II   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ABC TRANSPORTER; ABC TRANSPORTER A4; ALKALINE PHOSPHATASE; PARVOVIRUS VECTOR; RHODOPSIN KINASE; UNCLASSIFIED DRUG; ABCA4 PROTEIN, HUMAN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; FEMALE; GENE EXPRESSION; GENE THERAPY; HOMOLOGOUS RECOMBINATION; HUMAN; HUMAN CELL; INVERTED TERMINAL REPEAT; MOUSE; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN DEGRADATION; PROTEIN EXPRESSION; STARGARDT DISEASE; TRANSGENE; UBIQUITINATION; VIRAL GENE DELIVERY SYSTEM; ADMINISTRATION AND DOSAGE; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE VECTOR; GENETICS; HEK293 CELL LINE; INTRAOCULAR DRUG ADMINISTRATION; LONG TERMINAL REPEAT; MACULAR DEGENERATION; METABOLISM; PIG; PROCEDURES; RETINA;

ADMINISTRATION, OPHTHALMIC; ANIMALS; ATP-BINDING CASSETTE TRANSPORTERS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FEMALE; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; MACULAR DEGENERATION; MICE; RETINA; SWINE; TERMINAL REPEAT SEQUENCES; TRANSGENES;

EID: 84956706085     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv386     Document Type: Article

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