National Institute of Neurological Disorders and Stroke (NINDS)
Vectors for Gene Therapy in the Nervous System
October 23 – 24, 2000 • Bethesda, MD
Agenda
| Monday, October 23 |
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| 8:30 a.m. | Welcome Gerry Fischbach Workshop goals |
| 8:45 a.m. | Introductory remarks Rusty Gage/Inder Verma |
| Session 1: Gene therapy for Parkinson’s disease (PD) | |
| 9:00 a.m. | Overview of gene therapy for PD Un Kang |
| 9:30 a.m. | Research updates: Lentiviral gene delivery of GDNF for PD in primate models
Differential effects of GDNF gene delivery to the striatum
vs the substantia nigra New AAV production methodologies for CNS use |
| 10:15 a.m. | Discussion of technological issues:
1. Choice of gene 2. Choice of delivery method (vector, promoter, etc.) |
| 11:00 a.m. | Research updates: Dopaminergic recovery following an AAV/AADC approach in primates
Assessing the efficacy of gene therapy for PD: rat
behavioral models A new transgenic model of Parkinson’s disease |
| 11:30 a.m. | Discussion of clinical issues:
1. Patient selection 2. Monitoring gene expression 3. Outcome measures |
| Session 2: Gene therapy for lysosomal storage disorders (LSDs) | |
| 1:30 p.m. | Overview of the LSDs Sandra Hofmann
1. Similarities/differences among storage disorders
|
| 2:00 p.m. | Research updates:
Improved histopathology and cognitive function in a murine
model of LSD following gene therapy Palmitoyl-protein thioesterase and the molecular basis of infantile Batten
disease Gene therapy for global neurodegenerative disorders |
| 2:45 p.m. | Discussion of technological issues
1. Choice of gene 2. Choice of gene delivery method |
| 3:45 p.m. | Research updates:
Choosing mouse models for preclinical testing
Obstacles to gene therapy for lysosomal storage disorders
Issues in gene therapy for neurometabolic diseases |
| 4:30 p.m. | Discussion of clinical issues
1. Patient selection 2. Monitoring gene expression 3. Clinical outcome measures |
| Tuesday, October 24 | |
| Session 3: Research priorities and strategy recommendations | |
| 8:30 a.m. | General research updates:
Novel high-capacity adenoviruses: long-term transgene
expression in the presence of anti-adenoviral immunity
A lentivirus-based intercellular delivery method
The NCRR National Gene Vector Laboratories |
| 9:00 a.m. | Discussion: Priorities for future research efforts |
| 10:15 a.m. | Overview of strategic issues Bob Baughman |
| 10:30 a.m. | Gene therapy clinical trials—translation to the clinic Mark Tucyznski Gene therapy clinical trials—ethical and review issues FDA biological therapeutics approval procedure |
| 11:15 a.m. | Discussion: Strategy recommendations
1. Funding mechanisms 2. Possible collaborations (NGVLs, industry, etc.) 3. Regulatory approval |
| 12:00 p.m. | Concluding remarks Inder Verma/Rusty Gage |
Participants
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Krys S. Bankiewicz Molecular Therapeutic Section National Institute of Neurological Disorders and Stroke Building 36, Room 5W21 36 Convent Drive Bethesda, MD 20892 John A. Barranger Department of Human Genetics University of Pittsburgh E1652 Biomedical Science Tower Pittsburgh, PA 15261 Robert W. Baughman Technology Development National Institute of Neurological Disorders and Stroke NSC, Room 2137 6001 Executive Blvd. Rockville, MD 20892 Martha C. Bohn Molecular Pharmacology & Biological Chemistry Pediatrics Foundation 2300 Children's Plaza, Box 18 Chicago, IL 60611-HCMH Roscoe O. Brady Developmental and Metabolic Neurology Branch National Institute of Neurological Disorders and Stroke Building 10, Room 3D04 10 Center Drive, MSC 1260 Bethesda, MD 20892-1260 Ennio A. Chiocca Department of Surgery Massachusetts General Hospital Neurosurgical Service, White 502 Boston, MA 02114 Beverly L. Davidson Department of Internal Medicine University of Iowa 200 EMRB Iowa City, IA 52242 Matthew J. During CNS Gene Therapy Center Thomas Jefferson University Hospital 1025 Walnut Street College 511 Philadelphia, PA 19107 Howard J. Federoff Department of Neurology University of Rochester Medical Center 601 Elmwood Ave., Box 673 Rochester, NY 14642 Robert Finkelstein Neurogenetics National Institute of Neurological Disorders and Stroke NSC, Room 2143 6001 Executive Blvd. Rockville, MD 20892 Gerald Fischbach Director National Institute of Neurological Disorders and Stroke Building 31, Room 8A54 31 Center Drive Bethesda, MD 20892 Fred H. Gage Laboratory of Genetics Salk Institute for Biological Studies 10010 N. Torrey Pines Road La Jolla, California 92037 Sandra L. Hofmann Department of Internal Medicine Southwestern Medical Center University of Texas 5323 Harry Hines Blvd. Dallas, TX 75235-8593 Ole Isacson Neuroregeneration Laboratory McLean Hospital, Mailman Research Center 119 115 Mill Street Belmont, MA 02178 Eugene M. Johnson, Jr. Department of Neurology Washington University 4488 Forest Park Ave., Suite 130 St. Louis, MO 63108 Un Jung Kang Department of Neurology University of Chicago MC 2030 5841 South Maryland Ave. Chicago, IL 60637 Jeffrey H. Kordower Rush Neuroscience Institute Rush Presbyterian St. Luke’s Medical Center 1653 West Congress Parkway Chicago, IL 60612 Pedro R. Lowenstein Molecular Medicine and Gene Therapy Unit University of Manchester School of Medicine Stopford Building, Room 1.302 Oxford Road Manchester M13 9PT England Ronald Mandel Department of Neuroscience University of Florida College of Medicine P.O. Box 100244 Gainesville, FL 32610-0244 Anne Messer Wadsworth Center New York State Department of Health David Axelrod Institute of Public Health New Scotland Ave. P.O. Box 22002 Albany, NY 12201-2002 William C. Mobley Department of Neurology Stanford University 1201 Welch Rd. MSLS Room P211 Stanford, CA 94305-5489 Diane Murphy Neurodegeneration National Institute of Neurological Disorders and Stroke NSC, Room 2202 6001 Executive Blvd. Bethesda, MD 20892-9527 Philip D. Noguchi Division of Cellular & Gene Therapies Center for Biologics Evaluation and Research U.S. Food and Drug Administration Building N29B, Room 2NN20 9000 Rockville Pike Bethesda, MD 20892 Amy Patterson Office of Biotechnology Activities National Institutes of Health 6000 Executive Blvd. Rockville, MD 20892 Mark S. Sands Bone Marrow Transplant and Stem Cell Biology Washington University School of Medicine 660 South Euclid Ave. Box 8007 St. Louis, MO 63110 Giovanna Spinella Neurogenetics National Institute of Neurological Disorders and Stroke NSC, Room 2132 6001 Executive Blvd. Bethesda, MD 20892 Greg Stewart Genzyme 1 Mountain Rd. P.O. Box 9322 Framingham, MA 01701 Mark H. Tuszynski Center for Neural Repair University of California, San Diego Basic Science Building, Room 4046 9500 Gilman Drive La Jolla, CA 92093-0626 Inder M. Verma Laboratory of Genetics The Salk Institute for Biological Studies 10010 N. Torrey Pines Road La Jolla, California 92037 |
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