Melissa A. Calton

Design and characterization of 4D-710, an aerosolized gene therapy for cystic fibrosis lung disease

Melissa A. Calton, Roxanne H Croze, Sara Collins, Sarah C. Tucker et autres

Cystic fibrosis (CF) is an autosomal recessive disease caused by variants in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. Delivery of a functional CFTR transgene to airway epithelial cells (AEC) offers the potential to provide durable restoration of normal …

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Non-clinical evaluation of 4D-310 in combination with rituximab/sirolimus: A translational study to support adoption of a novel prophylactic immunomodulation regimen in clinical trials in adults with Fabry disease

Melissa A. Calton, Caralee J. Schaefer, Theodore R. Sullivan, Samantha N. Jensen et autres

Design and Characterization of a Novel Intravitreal Dual-Transgene Genetic Medicine for Neovascular Retinopathies

Melissa A. Calton, Roxanne H. Croze, Christian H. Burns, Ghezal Beliakoff et autres

Purpose: Intravitreal delivery of therapeutic transgenes to the retina via engineered viral vectors can provide sustained local concentrations of therapeutic proteins and thus potentially reduce the treatment burden and improve long-term vision outcomes for patients with neovascular (wet) age-related macular degeneration (AMD), …

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AMP-independent activator of AMPK for treatment of mitochondrial disorders

Tereza Moore, Rolando E. Yanes, Melissa A. Calton, Douglas Vollrath et autres

Mitochondrial diseases are a clinically heterogenous group of disorders caused by respiratory chain dysfunction and associated with progressive, multi-systemic phenotype. There is no effective treatment or cure, and no FDA-approved drug for treating mitochondrial disease. To identify and characterize potential therapeutic compounds, …

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Depletion of Mitochondrial DNA in Differentiated Retinal Pigment Epithelial Cells

Xinqian Hu, Melissa A. Calton, Shibo Tang, Douglas Vollrath

We investigated the effects of treating differentiated retinal pigment epithelial (RPE) cells with didanosine (ddI), which is associated with retinopathy in individuals with HIV/AIDS. We hypothesized that such treatment would cause depletion of mitochondrial DNA and provide insight into the consequences of …

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Highly Differentiated Human Fetal RPE Cultures Are Resistant to the Accumulation and Toxicity of Lipofuscin-Like Material

Qitao Zhang, Feriel Presswalla, Melissa A. Calton, Carol J. Charniga et autres

Purpose: The accumulation of undigestible autofluorescent material (UAM), termed lipofuscin in vivo, is a hallmark of aged RPE. Lipofuscin derives, in part, from the incomplete degradation of phagocytized photoreceptor outer segments (OS). Whether this accumulated waste is toxic is unclear. We therefore …

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Disease mechanisms elucidated by genetic regulation of human RPE gene expression

Douglas Vollrath, Boxiang Liu, Melissa A. Calton, Nathan S. Abell et autres

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VE-4840, an oral plasma kallikrein inhibitor, decreases human plasma kallikrein and VEGF-induced retinal thickening and vascular permeability

Melissa A. Calton, A A Jeffrey, Lev Igoudin, Sivan Sizikov et autres

Genetic analyses of human fetal retinal pigment epithelium gene expression suggest ocular disease mechanisms

Boxiang Liu, Melissa A. Calton, Nathan S. Abell, Gillie Benchorin et autres

Abstract The retinal pigment epithelium (RPE) serves vital roles in ocular development and retinal homeostasis but has limited representation in large-scale functional genomics datasets. Understanding how common human genetic variants affect RPE gene expression could elucidate the sources of phenotypic variability in …

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Ocular disease mechanisms elucidated by genetics of human fetal retinal pigment epithelium gene expression

Boxiang Liu, Melissa A. Calton, Nathan S. Abell, Gillie Benchorin et autres

Abstract The eye is an intricate organ with limited representation in large-scale functional genomics datasets. The retinal pigment epithelium (RPE) serves vital roles in ocular development and retinal homeostasis. We interrogated the genetics of gene expression of cultured human fetal RPE (fRPE) …

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An orally dosed plasma kallikrein inhibitor decreases retinal vascular permeability in a rat model of diabetic retinopathy

Melissa A. Calton, A Lieberman Jeffrey, Eric H. Chang, Jeffrey Litt et autres

Method for measuring extracellular flux from intact polarized epithelial monolayers.

Melissa A. Calton, Marielle O Beaulieu, Gillie Benchorin, Douglas Vollrath

Purpose: The Seahorse XFp platform is widely used for metabolic assessment of cultured cells. Current methods require replating of cells into specialized plates. This is problematic for certain cell types, such as primary human fetal RPE (hfRPE) cells, which must be cultured …

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