Francesca Simonelli
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
2008/04/27 by Albert M. Maguire, Francesca Simonelli, Eric A. Pierce +29 · 14 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Retinal Development and Disorders #Retinal Diseases and Treatments #Retinal and Macular Surgery
- Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
2009/12/01 by Francesca Simonelli, Albert M. Maguire, Francesco Testa +19 · 7 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Retinal Development and Disorders #Corneal Surgery and Treatments #Retinopathy of Prematurity Studies
- Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
2009/10/24 by Albert M. Maguire, Albert M Maguire, Katherine A High +45 · 5 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Ocular Disorders and Treatments #Retinal Development and Disorders #Retinal Diseases and Treatments
- The human visual cortex responds to gene therapy–mediated recovery of retinal function
2011/05/23 by Manzar Ashtari, Laura Cyckowski, Justin F. Monroe +8 · 1 citation
Biochemistry, Genetics and Molecular Biology · Medicine · #Retinal Development and Disorders #Retinal Diseases and Treatments #Connexins and lens biology
- Mutations in CTNNA1 cause butterfly-shaped pigment dystrophy and perturbed retinal pigment epithelium integrity
2015/12/21 by Nicole T M Saksens, Nicole T.M. Saksens, Mark P Krebs +38 · 1 citation
Biochemistry, Genetics and Molecular Biology · #Connexins and lens biology #Hippo pathway signaling and YAP/TAZ #Retinal Development and Disorders