2005 Young Investigator

Miguel Sena-Esteves, PhD

University of Massachusetts Medical School Worcester, MA
Associate Professor, Department of Neurology Gene Therapy Center


Research Focus: Oncogenesis
Cancer Type: Gene Therapy for Brain Cancer


Named 2011 Outstanding New Investigator by the American Society of Gene and Cell Therapy (ASGCT)

2005-2008 Research Grant:
This study is based on the genetic modification of normal brain cells to create an environment that prevents tumor growth. Adeno-associated virus [AAV] vectors will be used to introduce in normal brain cells, or in blood vessels, a gene that makes a protein that is selectively active against tumor cells. These experiments will investigate the efficiency of this therapy principle. These approaches may lead to the creation of widespread anti-tumor networks capable of preventing brain tumors from growing or appearing again after surgery.

Current Research:

Dr. Sena-Esteves is currently working on developing gene therapy treatments for various brain disorders. This includes not only cancer and brain tumors, but also Tay-Sachs and Sandhoff diseases. Through the use of AAV vectors, he has developed a method of delivering the missing enzymes contributing to Tay-Sachs and Sandhoff disease by injecting the vectors into specific parts of the body’s central nervous system (CNS). Results obtained from animal models have demonstrated promising results that hopefully will bring this treatment into a clinical trial for Tay-Sachs disease. Dr. Sena-Esteves is also developing new AAV vectors to better target brain tumors, as well as a new gene therapy treatment for Ataxia Telangiectasia. Also called Louis-Bar syndrome, Ataxia Telangiectasia is a neurological disorder that, beginning at a young age, impairs coordination of movement and motor functions, growth, and many other bodily functions. Dr. Sena-Esteves’ work has brought the world of gene therapy to a new forefront with promising implications for the future of both cancers of the brain, and other neurological disorders.

Return to Fellows List