Collaboration between Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. Clinical research in the areas of stem cell transplantation, cellular therapy, and gene therapy.

The Center fosters a multidisciplinary approach to new research as well as collaborative research endeavors in the area of gene therapy. The Vector Core manufactures several recombinant viral vectors.

The research is focused on various aspects of gene therapy, such as understanding basic virology, efficient gene delivery into the nucleus of cells, and incorporation of these genes into the genome.

Dr. Curiel heads the center and is engaged in advancing the application of gene transfer methods for treatment of human disease. The efforts are focused on the development of improved vectors to accomplish targeted, cell-specific delivery.

Research in the laboratory has centered on 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 Harvard Gene Therapy Initiative is headed by Dr. Richard Mulligan with the objective of promoting the use of gene therapy and to conduct research developing new gene delivery vector technologies.

Diseases of the lung, cardiovascular system, muscles, brain, and skin are focus areas of research as well as the development of gene therapy vectors and the identification of disease-causing genes.

The major goals of the Innovative Cancer Therapies Program will be to enhance communication and collaboration among investigators and to develop new funding opportunities.

The program has brought together regulatory, quality, product development, manufacturing and facilities engineering expertise to enable the translation of novel, experimental research into medicine for use in human clinical trials.

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.

The primary mission 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.

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.

Research focused on the creation of vector systems for appropriate delivery of therapeutic genes for treatment of cancer, inflammatory diseases and infectious diseases.

The research is on cardiovascular, neurodegenerative, and metabolism-related diseases. Main target genes are vascular growth factors, and LDL receptor in cardiovascular area and cytotoxic genes in cancer therapy.

Research covers a wide range of diseases along three main tracks: cellular gene therapy, viral vectors and non-viral vectors. The Institute is headed by Prof. Eithan Galun, M.D.

The group headed by Luigi Naldini performs basic research on gene transfer technologies and new gene therapy strategies for immunodeficiencies, lysosomal storage disorders, thalassemia, hemofilia B.

The Gene Therapy research group aims to design new anti-cancer therapies using oncolytic adenoviruses and modulation of gene expression, with particular attention to the p53 tumor suppressor pathway.

The department is headed by Prof. Hidde Haisma and is focused on the development of drugs for the therapeutic manipulation of gene activity. The research is focused on cancer and inflammatory diseases.

The laboratory investigates the properties of blood stem cells and is developing cell and gene therapy for blood disorders: Diamond-Blackfan Anemia and Gaucher Disease.

Research is focussed on a combination of cancer, cell biology, molecular medicine and immunology. Research includes retroviral receptors, AAV gene transfer, novel retrovirus packaging cell lines, hemopoietic and mesenchymal stem cells.

Research is focused on liver-targeted gene therapy, nerve and muscle diseases, Cystic Fibrosis and gene therapy for the prevention of malaria.