Engineering human chromosomes for gene therapy studies |
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| Authors: | Choo K H |
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| Affiliation: | 2. Department of Laboratory Medici, University of California, San Francisco, CAne;3. Howard Hughes Medical Institute, University of California, San Francisco, CA;1. Department Of Molecular Genetics And Biochemistry, University Of Pittsburgh, School Of Medicine, W1246 Biomedical Science Tower, Pittsburgh, Pa 15261, USA;1. The Salk Institute for Biological Studies, Laboratory of Genetics, PO Box 85800, San Diego, CA 92186-5800, USA;2. Laboratory of Genetics, The Salk Institute for Biological Studies, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA;1. Introgen Therapeutics, Inc., 2215 Holcombe Boulevard, Houston, TX 77030, USA;2. Section of Thoracic Molecular Oncology, Department of Thoracic and Cardiovascular Surgery, Box 445, The University of Texas of M.D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA |
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| Abstract: | We now have the capability to engineer human chromosomes that could be used to deliver therapeutic genes in gene therapy studies. These vectors have the advantages of being non-disruptive to the genome, non-immunogenic and are capable of carrying very large genes with all their regulatory sequences. What challenges lie ahead, and what future does this technology hold for gene therapy? |
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