A state of art cGMP core facility for manufacture and certification of AAV gene transfer vectors has been established at the Children's Hospital of Philadelphia.
Part of The Center for Cellular and Molecular Therapeutics, the Research Vector Core has expanded its capacity to provide infrastructure support for investigators interested in using viral vectors in their research model systems.
Human embryonic cells (hESCs) potentially provide an unlimited supply of normal and diseased tissues for basic research, cell-based therapies and drug screening assays.
CCMT hosts a number of research laboratories that focus on areas such as gene transfer therapy, immunology, stem cell and protein biochemistry. All faculty members work closely with each other in optimizing resource usage, ensuring academic excellence and clinical relevance.
|Learn About Our Research
Directed by Dr. Katherine High, an internationally renowned physician scientist in gene therapy for rare diseases, Center for Cellular and Molecular Therapeutics (CCMT) has been at the forefront of translational studies in multiple rare diseases.
Participate in a Clinical Study
Currently, our center sponsors two gene transfer Phase I/II studies: Hemophilia B and Leber's congenital amaurosis type 2(LCA2).
Contact UsJunwei Sun, MS, MBA Administrative Director