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The Grousbeck Gene Therapy Center is an academic research center devoted to the broadening of therapeutic gene transfer in modern medicine. Our focus is on internal and collaborative preclinical and clinical research studies, technology development, and enabling resources for the community.
Vandenberghe Lab
The Vandenberghe Lab is the Grousbeck Gene Therapy core laboratory focused on developing and translating genetic therapies to the clinic with an emphasis on blindness and other neurosensory disorders. Researchers in the Vandenberghe lab work at the intersection of medicine, virology, engineering, immunology, synthetic and systems biology to innovate genetic medicine.
Vector Core
The Gene Transfer Vector Core (GTVC) is to provide high quality research-grade viral vector preparations to support basic research and preclinical gene therapy studies for academia and industry. GTVC has its primary expertise in adeno-associated viral vector (AAV) preparation, qualification, and application.
News
November 13, 2019 Researchers Uncover Mechanism for How Common Gene Therapy Vectors Enter Cells
January 2, 2019 Nerea Zabalenta Lasarte, PhD joins the lab from the University of Navarra where she worked on liver-directed gene and genome editing therapies in the lab of Gloria González-Aseguinolaza.
September 2, 2018 Amanda Dudek graduates from the Harvard Virology Graduate Program on her work on AAV entry factors. Congratulations Amanda!
Upcoming Events
Trends and Innovations around the Treatment of Rare Disorders
Advances in Rare Disorders
A disorder is considered rare in the USA when it affects fewer than 200,000 people (or 1 in 2,000 in Europe). However, the scenario is of public concern if we consider the severity and the sheer number of these diseases. More than 6,000 rare disorders have been described affecting 300 million people around the world (30 million in Europe and 25 million in the USA), meaning that roughly one out of 15 people worldwide could suffer a rare disorder. Most of these diseases are chronic and life-threatening, and they present different challenges from those of more common diseases. The few patients that suffer from a rare disorder are usually scattered across countries, hampering the proper documentation of the natural history of the disease and patient management.
As a consequence, it takes an average of around five years to get a proper diagnosis, and 95% of rare disorders lack approved treatment. Besides, the impact on patients and families can be devastating, since 50% of the disorders affect children and 30% of them will die before the age of 5. The vast majority of rare diseases (80%) have a genetic component, and, therefore, the development of new drugs based on genetic information as well as gene and cell therapies is taking the lead on patient management. For this reason, and with the upcoming Rare Disease Day on February 29th, ECUSA would like to highlight the advances in cutting-edge treatments for these disorders.
Director
Luk H. Vandenberghe, PhD
Luk H. Vandenberghe, PhD, is an Associate Professor at Harvard Medical School and Associate Member of the Broad Institute of Harvard and MIT in Boston, MA, USA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, a part of the the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye.