Revised May 7, 2009

Ronald G. Crystal, MD, is the 2009 UAB Gene Therapy Center
Distinguished Guest Lecturer

(April 8, 2009)
Dr. Ronald G. Crystal was the 2008-2009 UAB Gene Therapy Center Seminar Series Distinguished Guest Lecturer. Dr. Crystal is Professor and Chairman of the Department of Genetic Medicine at the Weill Cornell Medical College in New York City. Dr. Crystal's group was the first to use a recombinant virus as a vehicle for in vivo gene therapy and has carried out human trials of gene therapy for cystic fibrosis, cardiac ischemia, cancer and central nervous system disorders. Among current projects are gene transfer strategies for cancer, inherited CNS disorders, a1-antitrypsin deficiency, anti-bioterrorism applications and development of vaccines. Dr. Crystal also operates the clinical vector production laboratory of the Belfer Gene Therapy Core Facility, which has produced adenovirus and adeno-associated virus vectors that have been used in numerous human studies. Current projects in the field of stem cell therapy include characterization of the roles of cancer stem cells in lung cancer and the role of airway epithelium stem cells in chronic obstructive pulmonary disease. Dr. Crystal's seminar on April 8th was entitled, "Genetic Medicines to Combat Bioterrorism". The presentation was co-sponsored by Vascular Biogenics, Ltd.

Oncologist Dr. Eva Galanis Visits the UAB Gene Therapy Center

(March 11, 2009)
Eva Galanis, M.D. recently visited the UAB Gene Therapy Center. Dr. Galanis is Professor in the Department of Oncology at the Mayo Clinic in Rochester, Minnesota, and is associated with the Gene and Virus Therapy Program at the Mayo Clinic. Her research centers on development of engineered measles virus for treatment of glioma, multiple myeloma and ovarian cancer, and has including Phase 1 clinical trials. Dr. Galanis' seminar on March 11th, 2009 was entitled "Oncolytic measles virus strains as cancer therapeutics: from the bench to the clinic" Dr. Galanis' presentation was co-sponsored by the UAB Comprehensive Cancer Center

AIDS Researcher John J. Rossi Visits UAB

(February 16, 2009)
The UAB Gene Therapy Center was very pleased to welcome Dr. John J. Rossi as a speaker in the 2008-09 Gene Therapy Center Seminar Series. John Rossi is Professor and Chair, Division of Molecular Biology at the Beckman Research Institute of the City of Hope National Medical Center in Duarte, CA. Dr. Rossi is a pioneer in the field of small RNA-based therapeutics, with applications for HIV/AIDS and cancer. Dr. Rossi's seminar on February 11th was entitled, "Small RNA based therapies for the treatment of HIV infection" Dr. Rossi's visit was co-sponsored by the UAB Center for AIDS Research (CFAR).

Hildegund C.J. Ertl Visits the UAB Gene Therapy Center

(January 28, 2009)
Dr. Hildegund Ertl recently visited the UAB Gene Therapy Center. Dr. Ertl is Professor and Program Leader of the Immunology Program at The Wistar Institute in Philadelphia, PA. Her research focus centers on vaccine development including HIV vaccines, human papilloma virus vaccines, rabies vaccine models, vaccines to smallpox virus, and vaccines to brain tumors Dr. Ertl's seminar on January 28th was entitled, "Immune responses to AAV vectors" Dr. Ertl's presentation was co-sponsored by the UAB Department of Microbiology.

Dr. Maria A. Croyle speaks at the UAB Gene Therapy Center

(December 11, 2008)
Dr. Maria A. Croyle's seminar on December 11th was entitled, "Pharmacology, Toxicology and Delivery Issues Associated with Recombinant Adenovirus-based Vaccines". Dr. Croyle is an Associate Professor in the Division of Pharmaceutics at the University of Texas at Austin College of Pharmacy.

Her research interests include Ad vector-based vaccine approaches, large-scale production methods for viral vectors for vaccines and gene therapy and the development of formulations that enhance vector stability as well as to develop methods that hide/mask recombinant viral vectors from the immune system and to predict how patients will react to a new gene medicine.

The presentation was co-sponsored by the UAB Department of Pharmacology and Toxicology.

2008-2009 GTC Seminar Series kicks off with Dr. Philip Ng

(Sept 25, 2008)
Dr. Philip Ng, Assistant Professor in the Department of Molecular and Human Genetics at Baylor College of Medicine was the first invited speaker of the 2008-2009 academic year. Dr. Ng's lab is developing gene therapies for genetic diseases using helper-dependent adenoviral vectors (HDAd). HDAd (also called gutless or gutted adenovirus) do not contain any viral genes and thus represent a major improvement over early generation adenoviral vectors with respect to safety and efficacy. A major focus of his laboratory is liver-directed gene therapy using HDAd to treat a wide variety of genetic disease such as hemophilia, Crigler-Najjar syndrome, cardiovascular disease, alpha 1-antitrypsin deficiency and many others. Another major focus is lung-directed gene therapy using HDAd with the primary goal of treating cystic fibrosis. Dr. Ng's seminar on September 24th was entitled, "Helper-dependent adenoviral vectors for gene therapy". This presentation was co-sponsored by the UAB Liver Center.

Gene Therapy Center Directors Meet in the Yucatan

(August, 2008)
Drs. David T. Curiel and J. Michael Mathis spent the week of July 28 - August 1 touring Mexico's Yucatan peninsula. Dr. Mathis is the Director of the Gene Therapy Program at LSU Health Sciences Center in Shreveport, LA, and David Curiel is the Director of the Gene Therapy Center and the Division of Human Gene Therapy at UAB.

Michael Mathis, David, Tasha and Nat Curiel stand in front of the 1000-year-old Mayan step pyramid of Chichen Itza, the Temple of Kukulcan, also known as El Castillo (the castle).

Dr. David T. Curiel (L) and Dr. Michael Mathis (R) display hard-won barracuda in Akumal, on the east coast of Quintana Roo.

Michel Sadelain, MD, PhD is the 2008
UAB Gene Therapy Center Distinguished Guest Lecturer

(May 20, 2008)
Dr. Michel Sadelain was the 2007-2008 UAB Gene Therapy Center Seminar Series Distinguished Guest Lecturer. Dr. Sadelain is a Member of the Department of Medicine and the Molecular Pharmacology Program at Memorial Sloan-Kettering Cancer Center, as well as the director of the Gene Transfer and Somatic Cell Engineering Center at the Sloan-Kettering Institute in New York, NY. Based on the use of recombinant oncoretro-viruses and lentiviruses, Dr. Sadelain's research group is investigating how to control transgene expression in vivo in hematopoietic stem cells and how to augment immune responses against tumor cells. Dr. Sadelain's seminar on May 20th was entitled, "Targeting tumors with genetically enhanced T-lymphocytes". The presentation was co-sponsored by Susan G. Komen for the Cure, North Alabama Affiliate.

Donald B. Kohn gives stem cell lecture
at UAB Gene Therapy Center

(May 6, 2008)
The UAB Gene Therapy Center welcomed pediatrician and researcher Donald B. Kohn, M.D. as a speaker in the UAB Gene Therapy Center Seminar Series. Dr. Kohn is Professor of Pediatrics in the Division of Research Immunology/Bone Marrow Transplantation at the Keck School of Medicine and Children's Hospital at the University of Southern California. His research efforts include the development of gene therapy methods for diseases of the hematopoietic and immunologic systems. Basic research includes the development of gene delivery vectors, focusing on methods to achieve more effective gene transfer into human hematopoietic stem cells and on more precise control of gene expression, in terms of level, lineage and longevity. Translational research efforts are on developing gene therapy approaches to genetic immune deficiency syndromes, HIV-1 and leukemia. Dr. Kohn's seminar on May 6th was entitled, "Gene Therapy Using Hematopoietic Stem Cells". The presentation was co-sponsored by the UAB Department of Genetics and Department of Pediatrics.

Dr. Tyler J. Curiel visits UAB, gives immunotherapy lecture

(L to R) David Curiel, Tyler Curiel and Laura Timares
UAB was pleased to welcome Dr. Tyler J. Curiel on March 3, 2008. Dr. Curiel is the Director of the Cancer Therapy & Research Center (CTRC) at the University of Texas Health Science Center at San Antonio. Dr. Tyler Curiel is a leading oncologist and is nationally known for his research in the areas of targeted cancer therapy and cancer immunotherapy. Importantly, Dr. Curiel's work predicts that reducing tumor-driven immune suppression will be clinically beneficial in the context of tumor immunotherapy. He highlights that the role of immunosuppressive regulatory T-cells, known, as T-regs, is one mechanism of tumor-driven immune evasion that may provide targets for testing novel anticancer treatment strategies. Dr. Curiel's seminar on March 3, 2008 was entitled "Regulatory T-cells as a Barrier to Effective Immune Therapy for Cancer" and was co-sponsored by the UAB Skin Diseases Research Center, Gene Therapy Center, and the Comprehensive Cancer Center.

Dr. Tyler Curiel brainstorms with members of the UAB Division of Human Gene Therapy

Dr. Christof von Kalle gives lecture in UAB Gene Therapy Seminar Series

Christof von Kalle, M.D. visited the UAB Gene Therapy Center on February 12, 2008. Dr. von Kalle is the Director of the National Center of Tumor Diseases (NCT) at the University Hospital Heidelberg and the German Cancer Center (DKFZ), Heidelberg, Germany. He is also the Chairman of the Department of Translation Oncology at the DKFZ, as well as Adjunct Professor of Pediatrics, Cincinnati Children's Hospital Medical Center, Division of Experimental Hematology, Cincinnati, OH. Dr. von Kalle is world-renowned for his research in the areas of gene therapy for human hemoglobinopathies, retrovirus-mediated therapy of hematopoietic stem cells, and clinical hematopoietic stem cell transplantation. Of note, von Kalle participated in the 1999 French gene therapy trial that restored the immune systems of nine of 11 boys with X-linked severe combined immunodeficiency disease (X-SCID), making this the first clear success in gene therapy. In 2003, Dr. von Kalle's group helped determine that in some cases the retrovirus engineered to deliver corrective genes into cells inserted itself in or near a cancer-causing gene, triggering leukemia in patients initially considered cured (see the initial report in Science). Dr. von Kalle's seminar on February 12th, 2008 was entitled "The clonal fate of gene modified blood cells".

American Society for Gene Therapy 10th Annual Meeting
Seattle, Washington
May 31-June 3, 2007

Members of the UAB Gene Therapy Center and UAB Division of Human Gene Therapy enjoyed the sights in and around Seattle while attending the 10th Annual Meeting of the American Society for Gene Therapy May 31-June 3, 2007 in Seattle, Washington. Twenty-six abstracts were presented by attendees from UAB.

James J. Cody presenting his work during a poster session

Anton V. Borovjagin (UAB) and Bruce F. Smith (Auburn University Vet School)

Joel Glasgow and UAB GTC Alumnus Koichi Takayama

Qiana L. Matthews at her poster

Yizhe Tang, Miho Murakami and Hideyo Ugai (L-R) at the ASGT 10th Anniversary Reception

Leaving Seattle; Mount St. Helens from the air. A horseshoe-shaped crater replaced the symmetrical peak after it's volcanic eruption in May, 1980. Over 400 meters of elevation was lost in 9 hours.

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