Gene Therapy
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85 slides .ppt
document in english
medical studies
presentation
date published 20/07/2007
review : not yet assessed
level : General public
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SummaryGene therapy is an experimental form of treatment whereby sequences of nucleic acids (i.e., genes) are delivered to cells to change their biologic function. The concept initially arose as replacement therapy for monogenic inherited disorders. For these disorders, the aim is to replace a defective gene with the normal counterpart. The delivered genetic material undergoes transcription and translation using the host cell's machinery, leading to in situ production of the normal protein and thereby correction of the phenotypic defect.
Table of Contents
- The Concept of Gene Therapy
- The Clinical Spectrum
- Gene Delivery Strategies
- THE DELIVERY VECTOR
- RETROVIRAL VECTORS.
- ADENOVIRAL VECTORS.
- ADENO-ASSOCIATED VIRUS
- Selected Disease Applications
- CYSTIC FIBROSIS
- HEMOPHILIA
- MYOCARDIAL AND PERIPHERAL ISCHEMIA
- HIV INFECTION AND AIDS
- REPLICATION-COMPETENT VIRUSES ("VIROTHERAPY")
- Recommendations
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