A5082358269- Retinal Development and Disorders
- Retinal Diseases and Treatments
- Cancer Research and Treatments
- Pleural and Pulmonary Diseases
- Drug-Induced Ocular Toxicity
- Ocular Disorders and Treatments
- Lung Cancer Research Studies
- Photoreceptor and optogenetics research
Penn Presbyterian Medical Center
2019-2022
University of Pennsylvania
2017-2022
Abstract CEP290-associated Leber congenital amaurosis type 10 (LCA10) is a retinal disease resulting in childhood blindness. Sepofarsen an RNA antisense oligonucleotide targeting the c.2991+1655A>G variant CEP290 gene to treat LCA10. In this open-label, phase 1b/2 ( NCT03140969 ), 12-month, multicenter, multiple-dose, dose-escalation trial, six adult patients and five pediatric received ≤4 doses of intravitreal sepofarsen into worse-seeing eye. The primary objective was evaluate safety...
Recessively-inherited NR2E3 gene mutations cause an unusual retinopathy with abnormally-increased short-wavelength sensitive cone (S-cone) function, in addition to reduced rod and long/middle-wavelength (L/M-cone) function. Progress toward clinical trials treat patients this otherwise incurable retinal degeneration prompted the need determine efficacy outcome measures. Comparisons were made between three computerized perimeters available clinic. These could deliver stimuli on...
Gene therapy for adRP due to RHO mutations was recently shown prevent photoreceptor death in a canine model of Class B disease. Among translational steps be taken, one is determine method detect efficacy human clinical trial. The relatively slow progression becomes difficulty trials requiring an answer whether there slowed degeneration response therapy. We performed single-center, retrospective observational study cross-sectional and longitudinal data. prompted by our identification...
Abstract Introduction: Malignant pleural effusions (MPE) occur as a frequent complication of advanced non-small cell lung cancer (NSCLC), and denote poor prognosis. Cytological evaluation MPEs has sensitivity only 40-60%. The CellSearch technology (Janssen Diagnostics, LLC) is an FDA approved method detecting rare circulating tumor cells (CTCs) in blood. Assessment fluid samples using provides approach to enrich often undetectable CTCs phenotype these for markers interest. PD-L1 staining...