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Volumn 374, Issue 9701, 2009, Pages 1569-1570

Promises and challenges of genetic therapy for blindness

Author keywords

[No Author keywords available]

Indexed keywords

EYE PROTEIN; PARVOVIRUS VECTOR; RETINAL PIGMENT EPITHELIUM 65 PROTEIN; UNCLASSIFIED DRUG;

EID: 70350619337     PISSN: 01406736     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0140-6736(09)61869-9     Document Type: Note
Times cited : (8)

References (12)
  • 1
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    • Effect of gene therapy on visual function in Leber's congenital amaurosis
    • Bainbridge J.W.B., Smith A.J., Barker S.S., et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358 (2008) 2231-2239
    • (2008) N Engl J Med , vol.358 , pp. 2231-2239
    • Bainbridge, J.W.B.1    Smith, A.J.2    Barker, S.S.3
  • 2
    • 44249085878 scopus 로고    scopus 로고
    • Safety and efficacy of gene transfer for Leber's congenital amaurosis
    • Maguire A.M., Simonelli F., Pierce E.A., et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358 (2008) 2240-2248
    • (2008) N Engl J Med , vol.358 , pp. 2240-2248
    • Maguire, A.M.1    Simonelli, F.2    Pierce, E.A.3
  • 3
    • 54449085219 scopus 로고    scopus 로고
    • Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
    • Cideciyan A.V., Aleman T.S., Boye S.L., et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA 105 (2008) 15112-15127
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 15112-15127
    • Cideciyan, A.V.1    Aleman, T.S.2    Boye, S.L.3
  • 4
    • 0041829006 scopus 로고    scopus 로고
    • A Tyr368His RPE65 founder mutation is associated with early-onset retinal dystrophy with variable expression and progression in 10 families of a genetically isolated population
    • Yzer S., van den Born L.I., Schuil J., et al. A Tyr368His RPE65 founder mutation is associated with early-onset retinal dystrophy with variable expression and progression in 10 families of a genetically isolated population. J Med Genet 40 (2003) 709-713
    • (2003) J Med Genet , vol.40 , pp. 709-713
    • Yzer, S.1    van den Born, L.I.2    Schuil, J.3
  • 5
    • 70350620424 scopus 로고    scopus 로고
    • Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
    • 10.1016/S0140-6736(09)61836-5 published online Oct 24.
    • Maguire A.M., High K.A., Auricchio A., et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet (2009) 10.1016/S0140-6736(09)61836-5 published online Oct 24.
    • (2009) Lancet
    • Maguire, A.M.1    High, K.A.2    Auricchio, A.3
  • 6
    • 10744230959 scopus 로고    scopus 로고
    • In utero gene therapy rescues vision in a murine model of congenital blindness
    • Dejneka N.S., Surace E.M., Aleman T.S., et al. In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther 9 (2004) 182-188
    • (2004) Mol Ther , vol.9 , pp. 182-188
    • Dejneka, N.S.1    Surace, E.M.2    Aleman, T.S.3
  • 7
    • 28444442243 scopus 로고    scopus 로고
    • Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
    • Acland G.M., Aguirre G.D., Bennett J., et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther 12 (2005) 1072-1082
    • (2005) Mol Ther , vol.12 , pp. 1072-1082
    • Acland, G.M.1    Aguirre, G.D.2    Bennett, J.3
  • 8
    • 68849088569 scopus 로고    scopus 로고
    • Vision 1 year after gene therapy for Leber's congenital amaurosis
    • Cideciyan A.V., Hauswirth W.W., Aleman T.S., et al. Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med 361 (2009) 725-727
    • (2009) N Engl J Med , vol.361 , pp. 725-727
    • Cideciyan, A.V.1    Hauswirth, W.W.2    Aleman, T.S.3
  • 10
    • 34247588271 scopus 로고    scopus 로고
    • PTC124 targets genetic disorders caused by nonsense mutations
    • Welch E.M., Barton E.R., Zhuo J., et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 447 (2007) 87-91
    • (2007) Nature , vol.447 , pp. 87-91
    • Welch, E.M.1    Barton, E.R.2    Zhuo, J.3
  • 11
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina S., Garrigue A., Wang G.P., et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118 (2008) 3132-3142
    • (2008) J Clin Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1    Garrigue, A.2    Wang, G.P.3
  • 12
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe S.J., Mansour M.R., Schwarzwaelder K., et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118 (2008) 3143-3150
    • (2008) J Clin Invest , vol.118 , pp. 3143-3150
    • Howe, S.J.1    Mansour, M.R.2    Schwarzwaelder, K.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.