Sarah Ursu
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View article: One IV HEDGES DNA vector administration encoding hGLA or hGH produces durable hGLA and hGH serum lvels in immunocompetent mice
One IV HEDGES DNA vector administration encoding hGLA or hGH produces durable hGLA and hGH serum lvels in immunocompetent mice Open
The great majority of human monogenic, single protein deficiency disease patients, who comprise ~ 0.5% of the population, are incurable. When available, Enzyme Replacement Therapy (ERT) is current state-of-the art therapy for the vast majo…
View article: Prolonged, Controllable Protein Production of cDNA-Encoded hGH Maintained at Therapeutic Serum Levels Following One Systemic Administration of a Non-Viral, Non-Integrating, DNA-and Liposome-Based <i>in vivo</i> Gene Therapy Platform in Immunocompetent Mice
Prolonged, Controllable Protein Production of cDNA-Encoded hGH Maintained at Therapeutic Serum Levels Following One Systemic Administration of a Non-Viral, Non-Integrating, DNA-and Liposome-Based <i>in vivo</i> Gene Therapy Platform in Immunocompetent Mice Open
Recombinant human growth hormone (rhGH), the mainstay of hGH replacement therapy, is injected daily for years to enable children to achieve normal stature. Daily rhGH injections are required because its serum-T1/2 is <20 minutes. Human …
View article: Durable multitransgene expression in vivo using systemic, nonviral DNA delivery
Durable multitransgene expression in vivo using systemic, nonviral DNA delivery Open
HEDGES, a tunable nonviral DNA-based gene therapy platform, durably expresses human therapeutic proteins in immunocompetent mice.
View article: Novel tumor suppressor role of miRNA-876 in cholangiocarcinoma
Novel tumor suppressor role of miRNA-876 in cholangiocarcinoma Open
Cholangiocarcinoma (CCA) is a rare, highly invasive malignancy, and its incidence is increasing globally. MicroRNAs (miRNAs) mediate a wide array of cellular and biological processes and are dysregulated in various tumors. The functional a…
View article: BPTF transduces MITF-driven prosurvival signals in melanoma cells
BPTF transduces MITF-driven prosurvival signals in melanoma cells Open
Significance Microphthalmia-associated transcription factor (MITF) is a master regulator of survival of the melanocyte lineage, exerting its effects through a cascade of transcriptional activation by interacting with a consensus DNA-bindin…
View article: Tumor suppressor role of microRNA-1296 in triple-negative breast cancer
Tumor suppressor role of microRNA-1296 in triple-negative breast cancer Open
Our results demonstrate a novel tumor suppressor role for miR-1296 in triple-negative breast cancer cell lines, identify CCND1 as its target of action, and demonstrate a potential role for miR-1296 in sensitizing breast cancer cells to cis…