Gene Therapy and Repair

.CIIT Carol Miao

The research of Carol Miao, PhD, is focused on developing gene therapy strategies to treat genetic diseases and immunomodulation protocols to overcome immune responses following gene or protein replacement therapy.

Center for Immunity and Immunotherapies investigators work on research programs supported by the National Institutes of Health (NIH) designed to develop clinical gene therapy protocols for X-linked agammaglobulinemia, Wiskott-Aldrich syndrome and hemophilia.

We launched the Program in Cell and Gene Therapy in 2014 as the first step in bringing molecular therapies to patients. This builds on our experience as the coordinating center for the Northwest Genome Engineering Consortium (NGEC), which brought together researchers at Seattle Children's Research Institute, Fred Hutchinson Cancer Research Center and the University of Washington to develop new methods for gene repair.

Led by Drs. Andy Scharenberg and David Rawlings, the NGEC was initiated by the National Institutes of Health (NIH) Roadmap for Medical Research, a new type of NIH grant program designed to address especially complex problems in research that require expertise across multiple scientific disciplines.

Read about the other innovative programs our center provides, including cellular therapeutics and molecular diagnostics.