- Center for Cell and Gene Therapy, Baylor College of Medicine, Houston
- Center for Gene Therapy, University of Michigan Medical Center, Ann Arbor
- Dep. of Gene and Cell Medicine, Mount Sinai School of Medicine, New York
- Gene Therapy Center, University of North Carolina School of Medicine
- Harvard Gene Therapy Initiative, Harvard University, Boston
- Iowa Center for Gene Therapy, University of Iowa, Iowa City
- Mayo Clinic Department of Molecular Medicine
- Mayo Graduate School, Mayo Clinic, Virology and Gene Therapy Track, Rochester
- Minnesota University, Molecular and Cellular Therapeutics Program
- Penn Vector Core, University of Pennsylvania, Philadelphia
- Powell Gene Therapy Center, University of Florida, Gainesville
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.
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.
A multidisciplinary team of scientists and physicians work together to realize the full potential of virus, gene and cell therapies from basic science discovery to clinical translation.
Oncolytic virotherapy, gene therapy for diabetes and cardiovascular diseases, virus-based gene therapy vectors.
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.
- Finland - A.I. Virtanen Institute of Molecular Medicine, University of Kuopio
- Finland - Cancer Gene Therapy Group, University of Helsinki
- Israel - The Goldyne Savad Institiute of Gene Therapy, Hadassah, Jerusalem
- Italy - San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Milan
- Italy - TIGEM - Telethon Institute of Genetics and Medicine, Napoli
- Netherlands - Oncogenomics VUmc, Amsterdam
- Netherlands - Pharmaceutical Gene Modulation, Groningen
- Spain - Viral Vector Production Unit (UPV), Universitat Autonoma de Barcelona
- Sweden - Molecular Medicine and Gene Therapy, Lund University
- Turkey - Human Gene and Cell Therapy Center, Akdeniz University Hospitals and Clinics
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.
The group of Akseli Hemminki uses gene therapy and oncolytic viruses to improve the treatment of cancers lacking currently available effective modalities.
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 interest of group of Alberto Auricchio is to use Adeno-Associated Virus (AAV)-mediated gene transfer as potential therapy for ocular and metabolic diseases
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.
UPV is a vector core specialized in the cloning, production and purification of adenovirus (human and canine) and adeno-associated virus (AAV) serotypes for public institutions and private companies all over the world.
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.
The laboratory is headed by Prof. Dr. Salih Sanlioglu and his team are involved in the development of novel gene therapy methods for patients with type 2 diabetes using lentiviral vectors.
- Cell and Molecular Therapies, Royal Prince Alfred Hospital, Camperdown
- Centenary Institute of Cancer Medicine and Cell Biology, Sydney
- Gene Silencing and Expression Facility (GSEx), Adelaide
- Gene Therapy Research Unit, Children's Medical Research Unit
HPC-A collection, processing, storage and transplant cGMP manufacturing of cell and gene modified human cells for clinical use.
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.
The Gene Silencing and Expression (GSEx) facility is a non-profit service lab providing viral vectors and other molecular services to researchers internal and external to the University of Adelaide.
Research is focused on liver-targeted gene therapy, nerve and muscle diseases, Cystic Fibrosis and gene therapy for the prevention of malaria.
FP6 is the European Community Framework Programme for Research, Technological Development and Demonstration. It is a collection of the actions at EU level to fund and promote research.
- Attack: Adoptive engineered T cell Targeting to Activate Cancer Killing, to improve T cell mediated immunotherapy to fight a broad range of cancer
- Clinigene: European Network for the Advancement of Clinical Gene Transfer and Therapy
- Consert: consortium of European research and clinical institutions cooperating to develop gene therapy for inherited diseases
- EPI-vector consortium to develop episomal vectors as gene delivery systems for therapeutic application
- GIANT: Gene Therapy, an integrated approach to neoplastic treatment, gene therapy for the treatment of prostate cancer
- Improved precision of Cystic Fibrosis therapy, a project investigating a new approach to the gene therapy of cystic fibrosis
- Tumor-Host Genomics, a concerted effort to understand tumor-host interactions, and to identify novel therapeutic targets
FP7 is the European Community Framework Programme for Research, Technological Development and Demonstration. FP7 will be fully operational as of 1 January 2007 and will expire in 2013. It is designed to build on the achievements of its predecessor towards the creation of the European Research Area, and carry it further towards the development of the knowledge economy and society in Europe. Please find below a selection of FP7 research projects on gene therapy.
- AAVEYE: Gene therapy for inherited severe photoreceptor diseases
- AIPGENE: Augmenting PBGD expression in the liver as a Novel Gene therapy for Acute Intermittent Porphyria
- BRAINCAV: Nonhuman adenovirus vectors for gene transfer to the brain
- CGT HEMOPHILIA A: Cell and gene therapy based strategies to correct the bleeding phenotype in Hemophilia A
- EYESEE: Development of new gene therapy approaches for the treatment of ocular neovascularization
- GENEGRAFT: Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector
- GENETHESIS: Gene Therapy: Modeling Synthetic DNA Delivery Systems
- IRLVGTMND: Improved retrograde lentiviral vectors for gene therapy in motor neuron diseases
- LGMD2A: Development of a strategy to treat limb-girdle muscular dystrophy (LGMD2A) using combined cell and gene therapy strategies
- NEUGENE: Advanced gene therapy tools for treatment of CNS-specific disorders
- ORAL-GT-VECTOR: Development of a Novel Vector for Cancer Gene Therapy for Clinical Application
- PERSIST: Persisting transgenesis Research area: HEALTH-2007-1.4-4 Development of emerging gene therapy tools and technologies for clinical application
- REGENERATIVETHERAPY: Cell and gene therapy approaches for inherited diseases with unsatisfying or no therapeutic option
- TARGETINGGENETHERAPY: Towards Safe and Effective Hematopoietic Stem Cell Gene Therapy: Targeting Integration to Genomic Safe Harbors and Exploiting Endogenous microRNA to Regulate Transgene Expression
- TREATPD: Cell and gene therapy based approaches for treatment of Parkinson's disease: From models to clinics
- VLPSIRNA: Virus-like particles: the next step in gene therapy