Paying for future success in gene therapy
| Autor: | Philip Reilly, Stuart H. Orkin |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty Multidisciplinary business.industry media_common.quotation_subject Genetic enhancement Treatment outcome Chronic pain Disease medicine.disease Genetic therapy 03 medical and health sciences 030104 developmental biology 0302 clinical medicine 030220 oncology & carcinogenesis medicine Anemia sickle-cell Girl Intensive care medicine business media_common |
| Zdroj: | Science. 352:1059-1061 |
| ISSN: | 1095-9203 0036-8075 |
| DOI: | 10.1126/science.aaf4770 |
| Popis: | Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited neurodegenerative disease. For decades, gene therapy has tantalized us with such futuristic scenarios. However, these goals are now coming into focus, and it is the time to consider some of the consequences of success. |
| Databáze: | OpenAIRE |
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