An incredible number and diversity of genetic diseases afflict humans. Despite the best efforts of medicine to reduce the suffering these diseases cause, curing them has remained out of reach. We are now at the point in time where genome engineering is stunningly close to being able to repair a genetic error and restore gene function. This volume outlines important examples of the tools and practice of gene therapy today. This incredible capability remains at the edges of existing science in new and predictably controversial territory. Similar to any other medical intervention, gene therapy is not devoid of risks. Understanding and controlling these risks while moving these technologies into the practice of medicine remain the goals for translational research. The chapters you are about to read demonstrate that fluency across basic science and clinical practice is possible and necessary for the robust application of gene therapy to improved survival.
|Original language||English (US)|
|Title of host publication||Translating Gene Therapy to the Clinic|
|Subtitle of host publication||Techniques and Approaches|
|Number of pages||10|
|State||Published - 2015|
Bibliographical noteFunding Information:
The author apologizes to the authors whose work could not be quoted because of space constraints and thanks Nancy Griggs Morgan for editorial assistance. J.T. is supported by grants from the National Institutes of Health (R01 AR063070 and R01 AR059947), the Department of Defense (USAMRAA/DOD Department of the Army W81XWH-12-1-0609 and USAMRAA/DOD W81XWH-10-1-0874), DebRA International, the Jackson Gabriel Silver Fund, the Epidermolysis Bullosa Medical Research Fund, University of Pennsylvania grant MPS I-11-009-01, and Children’s Cancer Research Fund, Minnesota.
© 2015 Elsevier Inc. All rights reserved..
Copyright 2020 Elsevier B.V., All rights reserved.
- Blood and marrow transplantation
- DNA structure
- Eusocial team
- Gene editing
- Gene therapy
- Immunological tolerance
- Nuclear transfer
- Selfish transgene
- Somatic mosaicism
- Stem cell therapy