My child has Leber congenital amaurosis: Why is he/she not eligible for gene therapy trials?

Journal of AAPOS

Volumn 13, Issue 6, 2009, Pages 533-534

  Héon, Elise ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AUTOSOMAL RECESSIVE INHERITANCE; EDITORIAL; GENE; GENE MUTATION; GENE THERAPY; GENETIC POLYMORPHISM; GENOTYPE; HUMAN; LEBER CONGENITAL AMAUROSIS; NONHUMAN; PHENOTYPE; PHYSICIAN; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RETINOID ISOMERASE GENE; RETINOPATHY;

CARRIER PROTEINS; CHILD; CHILD, PRESCHOOL; CLINICAL TRIALS AS TOPIC; EYE PROTEINS; FEMALE; GENE THERAPY; HUMANS; LEBER CONGENITAL AMAUROSIS; MALE; PATIENT SELECTION;

EID: 71649100132     PISSN: 10918531     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jaapos.2009.09.006     Document Type: Editorial

References (9)

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6. Jacobson S.G., Cideciyan A.V., Aleman T.S., et al. RDH12 and RPE65, visual cycle genes causing leber congenital amaurosis, differ in disease expression. Invest Ophthalmol Vis Sci 48 (2007) 332-338
7. Stone E.M. Leber congenital amaurosis---A model for efficient genetic testing of heterogeneous disorders: LXIV Edward Jackson Memorial Lecture. Am J Ophthalmol 144 (2007) 791-811
8. Jacobson S.G., Aleman T.S., Cideciyan A.V., et al. Defining the residual vision in leber congenital amaurosis caused by RPE65 mutations. Invest Ophthalmol Vis Sci 50 (2009) 2368-2375
9. Jacobson S.G., Aleman T.S., Cideciyan A.V., et al. Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success. Proc Natl Acad Sci USA 102 (2005) 6177-6182