Prominent Gene Therapy Expert Joins CHOP

Beverly L. Davidson, PhD, a nationally prominent expert in gene therapy, joined The Children’s Hospital of Philadelphia on April 1. A member of the Hospital’s Department of Pathology and Laboratory Medicine, Dr. Davidson 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. 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 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.

In addition to her roles at CHOP Dr. Davidson is a member of the Scientific Advisory Board of the Hereditary Disease Foundation, and is 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, and her team has succeeded in reversing neurological deficits in animal models of disease, and is working to advance this approach in humans.

She has also 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. 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.