TALEN-Mediated Gene Targeting for Cystic Fibrosis-Gene Therapy
Cystic fibrosis (CF) is an inherited monogenic disorder, amenable to gene-based therapies. Because CF lung disease is currently the major cause of mortality and morbidity, and the lung airway is readily accessible to gene delivery, the major CF gene therapy effort at present is directed to the lung....
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2019-01-01
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| Series: | Genes |
| Subjects: | |
| Online Access: | http://www.mdpi.com/2073-4425/10/1/39 |