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AN OPEN-LABEL DOSE ESCALATION STUDY OF AN ADENO-ASSOCIATED VIRUS VECTOR (AAV2/2-hRPE65p-hRPE65) FOR GENE THERAPY OF SEVERE EARLY-ONSET RETINAL DEGENERATION

Update Il y a 4 ans
Reference: EUCTR2006-001571-37

Woman and Man

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Extract

The main objective is to assess the safety and efficacy of a single subretinal administration of tgAAG76 in up to 12 patients with retinal dystrophy due to mutations in RPE65.

Inclusion criteria

  • The condition to be investigated is severe early-onset inherited retinal degeneration due to defects in the gene encoding RPE65

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