Medical Professional Publications

Innovation and Discovery

(From the March 2017 Issue of MedStat)

Visit NationwideChildrens.org/Research-News for additional information regarding these articles. More news from The Research Institute can be found at NationwideChildrens.org/Research-Now.

Researchers Investigate Disease Mechanisms that Allow Bacteria to Adhere to Molecules on Host Cell Surfaces, Critical in Heart Infection

Infective endocarditis (IE) is an infection of the endocardium, usually of the valves, and is caused by infectious agents that are typically bacterial. Understanding how these bacterial infections occur is important to the prevention, treatment and management of IE. A recent study from Nationwide Children’s Hospital is the first to demonstrate how a key protein is required to bind two distinct receptors on the bacteria, Streptococcus oralis, which is involved in IE. Samantha J. King, PhD, principal investigator in the Center for Microbial Pathogenesis in The Research Institute at Nationwide Children’s, is senior author of the study, which was published in the journal Infection.

Read the Research Now article.

Sarepta Therapeutics Enters Into Agreements with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program and Microdystrophin Gene Therapy Program

In January, Sarepta Therapeutics, Inc, a commercial stage developer for innovative RNA-targeted therapeutics, announced entering into a license agreement with Nationwide Children’s Hospital, for their Galgt2 gene therapy program, developed by Paul Martin, PhD, principal investigator in the Center for Gene Therapy in The Research Institute at Nationwide Children’s. Sarepta also announced entering into a research agreement with Nationwide Children’s on their microdystrophin gene therapy program, led by Jerry Mendell, MD, and Louise Rodino-Klapac, PhD, also principal investigators in the Center for Gene Therapy in The Research Institute at Nationwide Children’s.

Read News Room articles for the Galgt2 gene therapy program and the microdystrophin gene therapy program.

Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma

Two investigational agents, Aurora A kinase inhibitor (alisertib) and HSV1716, a virus derived from HSV-1 and attenuated by the deletion of RL1, have shown some antitumor efficacy in early clinical trials as monotherapies. A new study published in the journal Oncotarget, however, demonstrates that the combined usage of these agents results in significantly increased antitumor efficacy in models of malignant peripheral nerve sheath tumor (MPNST) and neuroblastoma. Timothy Cripe, MD, PhD, division chief of Hematology/Oncology & BMT at Nationwide Children’s Hospital, is senior author on the study, which was a collaboration among researchers from Nationwide Children’s, Cincinnati Children’s Hospital Medical Center, Takeda Pharmaceuticals International Co. and Virttu Biologics.

Read the Pediatrics Nationwide article.

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