Effective delivery of large genes to the retina by dual AAV vectors

EMBO Molecular Medicine

Volumn 6, Issue 2, 2014, Pages 194-211

  Trapani, Ivana ^a   Colella, Pasqualina ^a   Sommella, Andrea ^a   Iodice, Carolina ^a   Cesi, Giulia ^a   de Simone, Sonia ^a   Marrocco, Elena ^a   Rossi, Settimio ^b   Giunti, Massimo ^c   Palfi, Arpad ^d   Farrar, Gwyneth J ^d   Polishchuk, Roman ^a   Auricchio, Alberto ^a,e  

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

^b SECOND UNIVERSITY OF NAPLES   (Italy)

^c UNIVERSITY OF BOLOGNA   (Italy)

^d TRINITY COLLEGE DUBLIN   (Ireland)

^e UNIVERSITY OF NAPLES FEDERICO II   (Italy)

Author keywords

AAV; ABCA4; Gene therapy; MYO7A; Retina

Indexed keywords

ANIMALS; ATP-BINDING CASSETTE TRANSPORTERS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEK293 CELLS; HUMANS; INJECTIONS; LIPOFUSCIN; MACULAR DEGENERATION; MELANOSOMES; MICE; MICE, INBRED C57BL; MYOSINS; PHENOTYPE; PHOTORECEPTOR CELLS, VERTEBRATE; RETINA; RETINAL PIGMENT EPITHELIUM; RHODOPSIN; SUS SCROFA; TRANS-SPLICING; TRANSDUCTION, GENETIC; USHER SYNDROMES;

EID: 84893741157     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.1002/emmm.201302948     Document Type: Article

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