Academic Sites

North America

Center for Cell and Gene Therapy - Baylor College of Medicine
The Center for Cell and Gene Therapy is a collaborative effort between Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. The Center promotes the state-of-the-art clinical research in the areas of stem cell transplantation, cellular therapy, and gene therapy.

Centre for Gene Therapeutics - McMaster University
The mission of the Centre is to investigate, create and implement approaches utilizing the delivery of genes as therapeutic agents in the treatment of human and animal disease. This entails basic investigations to target gene product involvement, creation of vector systems for appropriate delivery of therapeutic genes and rapid translation of promising medicines to the clinical setting. The Centre is focused on developing novel cures for cancer, inflammatory diseases and infectious diseases using state-of-the-art gene transfer technology coupled with the most recent information derived from the application of genomics.

Center for Gene Therapy, Nationwide Children's Hospital - Columbus
Research at the Center for Gene Therapy includes molecular medicine: retrovirus; lentivirus, AAV, and adenovirus vectors metabolic disorders; HSC disorders; antiviral therapies, in utero and in vivo transfer; vectorology; large-scale production of vector; and animal model development.

Gene Therapy Center at University of Alabama at Birmingham
The Gene Therapy Center at UAB is engaged in advancing the application of gene transfer methods for treatment of human disease. Our efforts are focused on the development of improved vectors to accomplish targeted, cell-specific delivery, the training of physicians and scientists in the discipline of gene therapy and the rapid translation of rational gene therapy approaches into human clinical trials. In practice, pursuits in the context of the basic science of vectorology, gene therapy traineeship and clinical translation represents interlocking and cross-supportive disciplines. On this basis, the Gene Therapy Center functions as a true "center' that is the focal point for the advancement of gene therapy as a truly unique distinct discipline.

Gene Therapy Initiative: Harvard Institute of Human Genetics
The Harvard Gene Therapy Initiative was founded with the objective of promoting the use of gene therapy in both research and therapeutic applications and to conduct research developing new gene delivery vector technologies.

Institute of Gene Therapy and Molecular Medicine at Mount Sinai School of Medicine
The Institute of Gene Therapy and Molecular Medicine was inaugurated on September 1, 1996, for the specific purpose of expediting the scientific development of gene transfer and stem cell biology in facilitating their ultimate application in the treatment of human disease. Although the department's mission is to develop genes and cells as medicines to treat a wide variety of human diseases, the ultimate success in gene and cell therapeutics will be basic science driven. Some of the basic scientific advances generated from the department have led to the initiation of several Phase I clinical translational trials in gene therapy for various cancer targets. These federally-funded translational trials are being performed in close collaboration with the relevant clinical departments at Mount Sinai School of Medicine.

Virology and Gene Therapy Graduate Program at Mayo Clinic
The Virology and Gene Therapy Program offers a highly productive, interactive research environment in which to develop as an independent investigator. Enthusiastic faculty members have primary interests in virology, viral vectors and gene therapy. These areas overlap with the fields of biochemistry, cell and molecular biology, genetics and immunology.

Penn Vector
Penn Vector Core is an important technological resource for the University of Pennsylvania investigators and external investigators interested in the use of vectors for gene transfer. Penn Vector offers a variety of services associated with the development and production of both non-viral vectors and viral vectors including those derived from adeno-associated virus (AAV), adenovirus, and lentivirus. We provide a broad scope of services, extensive quality control and specialize in the development and production of novel serotype AAV vectors.

Powell Gene Therapy Center at the University of Florida
The primary mission of the Gene Therapy Center at the University of Florida is to merge molecular genetics research and health care delivery by developing new therapeutic strategies for the treatment of human diseases that involve gene transfer.

Program in Human Gene Therapy at University of California San Diego
The Program in Human Gene Therapy at UCSD, directed by Theodore Friedmann, MD, serves as the focus for gene therapy activities on the UCSD campus. The program coordinates gene therapy studies among the many investigators carrying out gene transfer and gene therapy studies at UCSD through vector production, research collaborations, grant preparation, lectures and seminar presentations, public relations and educational activities in gene therapy. The program includes a vector production core facility that assists investigators at UCSD and other institutions in the design and production of research grade vectors. The program also provides efficient and smooth transition to GMP vector production, clinical application and assistance with regulatory compliance through interactions with Molecular Medicine, LLC, an independent company derived from the gene therapy program and situated on the UCSD campus. The licensed facility is jointly operated by the University of California and Hoffman La Roche Pharmaceuticals.

Tulane Regional Primate Research Center
The Tulane Regional Primate Research Center is interested in collaborative projects that utilize rhesus monkeys.

Tulane University Center for Gene Therapy
The Center performs research characterizing adult stem cells, called mesenchymal stem cells or multipotential stromal cells (MSC) with a goal of using autologous adult stem cells for treating a variety of diseases. The Center is a NIH-funded center for distribution of well characterized human and rodent MSC to academic researchers worldwide. The Center has a cGMP laboratory comprising four production rooms capable of producing MSC for clinical trials.

Further information about the Center may be found at: http://www.som.tulane.edu/gene_therapy/

University of Arkansas for Medical Science
University of Arkansas for Medical Science (UAMS) is located in Little Rock. The ACRC (Arkansas Cancer Research Center) affiliated to UAMS has both strong research and clinical programs. The center has been performing multiple clinical gene therapy trials for industry and is looking for more trial opportunities. There are at least four laboratories dedicated to basic research and translational research for gene therapy. The research focus for cancer gene therapy includes adenoassociated viral gene delivery, electroporation gene delivery, cancer cell-targeted gene delivery, and molecular mechanism of gene therapy.

University of Michigan Center for Gene Therapy
The University of Michigan Center for Gene Therapy was created to link basic science, clinical investigation and technology transfer and to foster a multidisciplinary approach to collaborative basic and clinical gene therapy research endeavors.

University of North Carolina Chapel Hill
Research in the laboratory at the University of North Carolina Chapel Hill has centered around the study of the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy. The unique features that make this virus attractive for human gene therapy include the facts that it is able to insert its genome locus-specifically into human chromosome 19 and that is has never been identified as a causative agent of human disease. The locus specific integration is a valuable feature, as the risks of mutagenesis due to random insertions (as those associated with other gene transfer vectors) are potentially minimized. Continued efforts in understanding the mechanism of viral replication and integration for both wild-type and recombinant AAV are being pursued in order to create more efficient gene transfer vectors.

University of Pittsburgh Department of Microbiology and Molecular Genetics
The Department's research mission includes the discovery of fundamental mechanisms of microbial pathogenesis and the host response to infection, improve understanding of cell cycle biology, signaling pathways, DNA damage and repair and the development of cancer, uncover molecular mechanisms of tissue and organ development, explore the organization and function of various genomes and develop methods for translation of basic research to treatments of human disease.

University of Texas Medical Branch
Gene therapy for Canvan Disease. A mouse model is now available. The following vectors will be used - AAV, adenovirus, neuronal stem cells.

Waisman Clinical BioManufacturing Facility
The primary mission of the Waisman Clinical BioManufacturing Facility(WCBF) is to serve academic, government, and industry researchers as they enter development of novel biological therapies and to catalyze the advancement of therapies into human clinical trials. The WCBF is a dedicated manufacturing facility that provides cGMP manufacturing services for producing plasmid DNA, viral vectors, recombinant proteins, and cell therapeutics for early-stage human clinical trials. Services that are provided by the WCBF range from process development to clinical production and product fill and finish, all with full quality system support.

W.M. Keck Center for Cancer Gene Therapy

Europe

A.I. Virtanen Institute of Molecular Medicine at the University of Kuopio
A.I. Virtanen Institute of Molecular Medicine at the University of Kuopio, Finland, has focused its research on gene therapy of cardiovascular diseases (ischemia, in-stent restenosis and familial hypercholesterolemia) and malignant glioma gene therapy. In both areas Institute's programs have led to human phase II/III clinical trials. Also, the Institute has fully approved GMP laboratory and excellent large animals and imaging facilities which significantly contribute to the gene therapy programs. Main target genes are members of the VEGF family, other vascular growth factors, and LDL receptor in cardiovascular area and cytotoxic genes in cancer therapy.

Max Delbrück Center for Molecular Medicine Berlin Buch - Medical Genetics
German information site, offering information on gene transfer and gene therapy in cancer, cardiovascular system, and liver.

Asia and the Pacific Rim

The Gene and Stem Cell Therapy Program of the Centenary Institute
The Gene and Stem Cell Therapy Program of the Centenary Institute of Cancer Medicine and Cell Biology in Sydney, Australia headed by Professor John Rasko studies: retroviral receptors, system AAV gene transfer, novel retrovirus packaging cell lines, hemopoietic and mesenchymal stem cells.

Gene Therapy Research Unit, Children's Hospital at Westmead and Children's Medical Research Institute Australia
The unit focuses on the development and translation of gene therapy for acquired and heritable diseases of childhood in a strong clinical and research environment.