• Huntington's Disease
• Background Information
• Process of transcription
  and translation
• Gene therapy
• Virtual Lab 1: Polymerase
  Chain Reaction
• Revision Questions
• 

Gene therapy: Introduction

Gene therapy 基因治療 makes use of genes to treat a disease, instead of using therapeutic drugs. In principle, genetic diseases can be treated by gene therapy in one of the following ways:

1. Replacing the damaged gene with the normal gene
2. Inserting a gene that helps fighting against the disease
3. Introducing healthy copies of the damaged genes into the genome

To treat a disease using gene therapy, the following must be considered:

1. The pathology 病理 of the disease is known
2. The gene responsible for the disease is identified
3. The tissue or cell targeted is accessible
4. Method of introducing the gene into the cell is available, either ex vivo or in vivo
   1. Ex vivo gene therapy - modifying genes outside the body and put them back into body.
   2. In vivo gene therapy - modifying gene inside the body by transferring vector with normal gene into the body directly.
5. The procedure does not harm the patient.
6. Treatment can significantly improve the health of the patient.

Please see the following animation for the procedure of gene therapy.