Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex vivo

Gene Therapy

Volumn 23, Issue 11, 2016, Pages 767-774

  De Silva, S R ^a   Charbel Issa, P ^a,b   Singh, M S ^a   Lipinski, D M ^a   Barnea Cramer, A O ^a   Walker, N J ^a   Barnard, A R ^a   Hankins, M W ^a   MacLaren, R E ^a,c  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b UNIVERSITY OF BONN   (Germany)

^c MOORFIELDS EYE HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR; PHENYLALANINE; TYROSINE; ABC TRANSPORTER; ABCA4 PROTEIN, MOUSE; CAPSID PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; EX VIVO GENE TRANSFER; EXPRESSION VECTOR; HISTOLOGY; HUMAN; IN VIVO STUDY; MOUSE; MUTATION; NONHUMAN; PHOTORECEPTOR; PRIORITY JOURNAL; RETINA CELL; RETINA DEGENERATION; STARGARDT DISEASE; TROPISM; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID; ADMINISTRATION AND DOSAGE; ANIMAL; C57BL MOUSE; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; INTRAVITREAL DRUG ADMINISTRATION; METABOLISM; MISSENSE MUTATION; PHOTORECEPTOR CELL; RETINAL DEGENERATION; TUMOR CELL LINE;

ANIMALS; ATP-BINDING CASSETTE TRANSPORTERS; CAPSID PROTEINS; CELL LINE, TUMOR; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INTRAVITREAL INJECTIONS; MICE; MICE, INBRED C57BL; MUTATION, MISSENSE; PHOTORECEPTOR CELLS; RETINAL DEGENERATION;

EID: 84981508189     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2016.54     Document Type: Article

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