Term
| When the first gene therapy was approved?What for? |
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Definition
| 2012, Glybera, lipoprotein lipase deficiency |
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Term
| What are the purposes of gene therapy? |
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Definition
1) Compensation of a mutated gene
2) Introducing a new, engineered gene into the body to help fight a disease . |
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Term
| What are the main features of gene delivery via plasmids? |
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Definition
1) Low immunogenicity
2) Large capacity
3) No accessory machinery
4) No active cell uptake
5) Short term
6) No cell specificity |
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Term
| What are the main features of gene delivery via cationic lipids? |
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Definition
1) Cell permeability
2) No active cell uptake
3) Short term
4) No target cell |
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Term
| What are the main challenges of gene therapy? |
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Definition
• long-term efficacy
• Oncogenic |
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Term
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Definition
| Ex vivo - extract cells from patient, treat, return back |
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Term
| How gene therapy can be used in cancer? |
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Definition
1) Chimeric Antigen Receptor (CAR) T-Cell Therapy
2) T-cell extraction, T cell transfection, T-cell return to organism |
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Term
| How gene therapy could be applied infectious diseases? |
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Definition
| HIV and the discovery of broadly neutralizing antibodies (bnAbs) |
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Term
| Name the systems for genome editing |
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Definition
-Zinc-Finger Nucleases (ZFN)
-TALENs
- CRISPR/Cas |
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Term
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Definition
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