权威例句
Leber congenital amaurosis: genes, proteins and disease mechanisms.Safety and efficacy of gene transfer for Leber's congenital amaurosisEffect of gene therapy on visual function in Leber's congenital amaurosis.Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector AdministrationReversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.Leber congenital amaurosis: Comprehensive survey of the genetic heterogeneity, refinement of the clinical definition, and genotype&n...Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector:...Impairment of the Transient Pupillary Light Reflex in Rpe65(-/-) Mice and Humans with Leber Congenital AmaurosisMutations in the RPE65 Gene in Patients with Autosomal Recessive Retinitis Pigmentosa or Leber Congenital Amaurosis