April 2014

Gene Therapist Beverly Davidson, PhD, Joins CHOP


Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1. She joins the Hospital’s Department of Pathology and Laboratory Medicine and will serve as the new director of the Center for Cellular and Molecular Therapeutics.

Prior to coming to CHOP, Dr. Davidson, who investigates gene therapy for neurodegenerative diseases, was associate director of the Center for Gene Therapy at the University of Iowa. She also served as director of the University of Iowa’s Gene Therapy Vector Core, and held the Roy J. Carver Biomedical Research Chair in Internal Medicine.

Dr. Davidson “will greatly enhance our abilities to translate important biological discoveries into pioneering treatments for deadly diseases,” said Robert W. Doms, MD, PhD, CHOP’s pathologist-in-chief. Dr. Davidson has been named to Children’s Hospital’s Arthur V. Meigs Chair in Pediatrics.

She has also assumed the role of director of the Center for Cellular and Molecular Therapeutics (CCMT), succeeding the Center’s inaugural director, Katherine A. High, MD. The CCMT is “dedicated to the understanding, development, and application of gene and related cell and nucleic acid therapies and the promotion of professional public education,” according to its website.

“I am thrilled that we have been able to recruit one of the premier translational investigators in the U.S. to serve as the next director of the Center. I have led the Center for the last ten years, and I eagerly look forward to the innovations of the next decade, under Dr. Davidson’s leadership,” said Dr. High.

In addition to her roles at CHOP Dr. Davidson is currently a member of the Scientific Advisory Board of the Hereditary Disease Foundation, and is Chair of Medical Sciences of the American Association of the Advancement of Science. She is also a scientific co-founder and advisor at the gene therapy company Spark Therapeutics, which launched in late 2013 with a $50 million capital commitment from CHOP.

Research Interests

Dr. Davidson’s research has been concentrated on inherited genetic diseases that attack the central nervous system, with a particular focus on childhood-onset neurodegenerative diseases such as Batten disease and similar disorders.  According to the NIH, Batten disease and related disorders affect “an estimated 2 to 4 of every 100,000 live births in the United States.”

In these disorders, the lack of an enzyme impairs lysosomes, proteins that perform crucial roles in removing unwanted by-products of cellular metabolism. Toxic waste products then accumulate in the brain and cause progressively severe brain damage. Dr. Davidson has developed novel methods to deliver therapeutic genes to the central nervous system. Her laboratory team has succeeded in reversing neurological deficits in small and large animal models of disease, and is working to advance this approach to treating human diseases.

In addition to lysosomal storage disorders, she has studied other inherited neurological diseases such as Huntington’s disease and spino-cerebellar ataxia. In these studies, she has delivered forms of RNA to the brains of animals to silence the activity of disease-causing genes. Some of Dr. Davidson’s recently published papers include a Neuron paper on microRNA binding sites in the brain, a Human Molecular Genetics study on functional microRNAs and target sites, and a paper published in Neurotherapeutics on RNA interference therapeutics.

Although much of Dr. Davidson’s work has centered on delivering beneficial genes to the central nervous system, the viral vectors that she has developed are applicable to other organs and tissues — for example, in gene therapy directed to the lungs or the liver.

For more information about Dr. Davidson and her research, see the Hospital’s press release about her appointment. To learn more about the pioneering gene therapy research being performed at Children’s Hospital, see the CCMT site.

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