Opinion paper on the current status of the regulation of gene therapy in Europe
The potential applications of gene therapy are many, extending from monogenic hereditary diseases to acquired and multifactorial disorders. Therapeutic gene transfer addressing such a variety of conditions is currently being investigated, creating therapeutic options for diseases where none had prev...
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| Veröffentlicht in: | Human gene therapy 2002-11, Vol.13 (17), p.2085-2110 |
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| container_title | Human gene therapy |
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| creator | COHEN-HAGUENAUER, Odile ROSENTHAL, Felicia THIELEMANS, Kris VILE, Richard ZWIERZINA, Heinz CICHUTEK, Klaus GÄNSBACHER, Bernd BOLHUIS, Reinder DORSCH-HÄSLER, Karoline ESHHAR, Zelig GAHRTON, Gösta HOKLAND, Peter MELANI, Cecilia RANKIN, Elaine |
| description | The potential applications of gene therapy are many, extending from monogenic hereditary diseases to acquired and multifactorial disorders. Therapeutic gene transfer addressing such a variety of conditions is currently being investigated, creating therapeutic options for diseases where none had previously been available. The field of gene therapy represents one of the most challenging therapeutics for the new millennium. As such the concept might have been oversold ahead of its time. Nevertheless, is some instances, potential clinical efficacy is currently being found with reports of significant successes, for example, in inherited severe combined immunodeficiency disorders (SCID), hemophilia, arteritis obliterans, and even cancer. The development of recombinant DNA technology has caused fear in the public and speculation regarding its potential risks. Public reaction to the implementation of gene therapy is at best ambivalent, ranging from enthusiasm about its formidable therapeutic promises to apprehension concerning its putative harmful consequences. Indeed, the report of accidents in the United States resulted in a broad debate on the subject of gene therapy regulation brought to the attention of the U.S. Senate and Government. In addition, the recent occurrence of a malignant proliferation of the hematopoietic system in one gene therapy-treated SCID patient has been reported (www.astg.org). with a monoclonal insertion of the retrovirus vector at a sensitive locus (LMO2). |
| doi_str_mv | 10.1089/10430340260395938 |
| format | Article |
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Therapeutic gene transfer addressing such a variety of conditions is currently being investigated, creating therapeutic options for diseases where none had previously been available. The field of gene therapy represents one of the most challenging therapeutics for the new millennium. As such the concept might have been oversold ahead of its time. Nevertheless, is some instances, potential clinical efficacy is currently being found with reports of significant successes, for example, in inherited severe combined immunodeficiency disorders (SCID), hemophilia, arteritis obliterans, and even cancer. The development of recombinant DNA technology has caused fear in the public and speculation regarding its potential risks. Public reaction to the implementation of gene therapy is at best ambivalent, ranging from enthusiasm about its formidable therapeutic promises to apprehension concerning its putative harmful consequences. Indeed, the report of accidents in the United States resulted in a broad debate on the subject of gene therapy regulation brought to the attention of the U.S. Senate and Government. In addition, the recent occurrence of a malignant proliferation of the hematopoietic system in one gene therapy-treated SCID patient has been reported (www.astg.org). with a monoclonal insertion of the retrovirus vector at a sensitive locus (LMO2).</description><identifier>ISSN: 1043-0342</identifier><identifier>EISSN: 1557-7422</identifier><identifier>DOI: 10.1089/10430340260395938</identifier><identifier>PMID: 12490003</identifier><identifier>CODEN: HGTHE3</identifier><language>eng</language><publisher>Larchmont, NY: Liebert</publisher><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Applied cell therapy and gene therapy ; Bioethics ; Biological and medical sciences ; Biotechnology ; Clinical Trials as Topic - legislation & jurisprudence ; Europe ; European Union ; Fundamental and applied biological sciences. Psychology ; Gene therapy ; Gene Transfer Techniques - standards ; Gene Transfer Techniques - trends ; Genetic Therapy - legislation & jurisprudence ; Genetic Therapy - trends ; Health. Pharmaceutical industry ; Humans ; Industrial applications and implications. Economical aspects ; Medical sciences ; Transfusions. Complications. Transfusion reactions. 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Therapeutic gene transfer addressing such a variety of conditions is currently being investigated, creating therapeutic options for diseases where none had previously been available. The field of gene therapy represents one of the most challenging therapeutics for the new millennium. As such the concept might have been oversold ahead of its time. Nevertheless, is some instances, potential clinical efficacy is currently being found with reports of significant successes, for example, in inherited severe combined immunodeficiency disorders (SCID), hemophilia, arteritis obliterans, and even cancer. The development of recombinant DNA technology has caused fear in the public and speculation regarding its potential risks. Public reaction to the implementation of gene therapy is at best ambivalent, ranging from enthusiasm about its formidable therapeutic promises to apprehension concerning its putative harmful consequences. Indeed, the report of accidents in the United States resulted in a broad debate on the subject of gene therapy regulation brought to the attention of the U.S. Senate and Government. In addition, the recent occurrence of a malignant proliferation of the hematopoietic system in one gene therapy-treated SCID patient has been reported (www.astg.org). with a monoclonal insertion of the retrovirus vector at a sensitive locus (LMO2).</description><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Applied cell therapy and gene therapy</subject><subject>Bioethics</subject><subject>Biological and medical sciences</subject><subject>Biotechnology</subject><subject>Clinical Trials as Topic - legislation & jurisprudence</subject><subject>Europe</subject><subject>European Union</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Gene therapy</subject><subject>Gene Transfer Techniques - standards</subject><subject>Gene Transfer Techniques - trends</subject><subject>Genetic Therapy - legislation & jurisprudence</subject><subject>Genetic Therapy - trends</subject><subject>Health. Pharmaceutical industry</subject><subject>Humans</subject><subject>Industrial applications and implications. Economical aspects</subject><subject>Medical sciences</subject><subject>Transfusions. Complications. Transfusion reactions. 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| ispartof | Human gene therapy, 2002-11, Vol.13 (17), p.2085-2110 |
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| source | Mary Ann Liebert Online Subscription; MEDLINE |
| subjects | Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Applied cell therapy and gene therapy Bioethics Biological and medical sciences Biotechnology Clinical Trials as Topic - legislation & jurisprudence Europe European Union Fundamental and applied biological sciences. Psychology Gene therapy Gene Transfer Techniques - standards Gene Transfer Techniques - trends Genetic Therapy - legislation & jurisprudence Genetic Therapy - trends Health. Pharmaceutical industry Humans Industrial applications and implications. Economical aspects Medical sciences Transfusions. Complications. Transfusion reactions. Cell and gene therapy |
| title | Opinion paper on the current status of the regulation of gene therapy in Europe |
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