Fighting blindness with adeno-associated virus serotype 8

Human Gene Therapy

Volumn 22, Issue 10, 2011, Pages 1169-1170

  Auricchio, Alberto ^a,b  

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

^b UNIVERSITY OF NAPLES FEDERICO II   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 8; BLINDNESS; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; HUMAN; INHERITED RETINAL DEGENERATION; LEBER CONGENITAL AMAUROSIS; NONHUMAN; NOTE; PHOTORECEPTOR; PIGMENT EPITHELIUM; RETINA DEGENERATION; RETINITIS PIGMENTOSA; SEROTYPE; VISION;

ANIMALS; GENE THERAPY; GUANYLATE CYCLASE; LEBER CONGENITAL AMAUROSIS; PHOTORECEPTOR CELLS, VERTEBRATE; RECEPTORS, CELL SURFACE; VISION, OCULAR;

ADENO-ASSOCIATED VIRUS;

EID: 80155139457     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.2521     Document Type: Note

References (7)

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2. Boye, S.E., Boye, S.L., Pang, J., et al. (2010). Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One 5, e11306.
3. Boye, S.L., Conlon, T., Erger, K., et al. (2011). Long term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Invest. Ophthalmol. Vis. Sci. 52, 7098-7108.
4. Mihelec, M., Pearson, R.A., Robbie, S.J., et al. (2011). Long-term preservation of Cones and Improvement in Visual function following gene therapy in a mouse model of Leber congenital amaurosis caused by guanylate cyclase-1 deficiency. Hum. Gene Ther. 22, 1179-1190.
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6. Tan, M.H., Smith, A.J., Pawlyk, B., et al. (2009). Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: Effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. Hum. Mol. Genet. 18, 2099-2114.
7. Vandenberghe, L.H., Bell, P., Maguire, A.M., et al. (2011). Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci. Transl. Med. 3, 88ra54.