What is the primary objective of gene therapy?a)To replace healthy gen...
- Gene therapy aims to treat genetic diseases by introducing normal genes into an individual to compensate for the non-functional or defective genes.
- This can potentially cure genetic disorders.
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What is the primary objective of gene therapy?a)To replace healthy gen...
Primary Objective of Gene Therapy: To cure genetic diseases using normal genes.
Gene therapy is a medical technique that involves the introduction or alteration of genetic material within a person's cells to treat or prevent a disease. The primary objective of gene therapy is to cure genetic diseases by replacing or repairing defective genes with normal genes.
1. Treating Genetic Diseases:
Gene therapy aims to treat genetic diseases that are caused by mutations or abnormalities in a person's genes. These diseases can be inherited from parents or acquired due to genetic mutations. By introducing normal genes into the affected cells, gene therapy seeks to correct the underlying genetic cause of the disease and restore normal cellular function.
2. Replacing Defective Genes:
One approach in gene therapy involves replacing faulty genes with normal copies of the gene. This can be achieved by delivering the normal gene into the patient's cells using various methods such as viral vectors, liposomes, or gene editing technologies like CRISPR-Cas9. Once the normal gene is inserted, it can produce functional proteins, enzymes, or other molecules that were previously lacking due to the defective gene.
3. Repairing Mutated Genes:
Another approach in gene therapy focuses on repairing mutated genes within the patient's cells. This can be done using techniques like gene editing, where specific changes are made to the DNA sequence to correct the mutation. By repairing the mutated gene, gene therapy aims to restore normal gene function and alleviate the symptoms of the genetic disease.
4. Preventing Genetic Diseases:
In some cases, gene therapy can also be used to prevent the occurrence of genetic diseases. This can be achieved by introducing normal genes into the cells of individuals who have a high risk of developing a particular genetic condition. By providing the correct genetic instructions, gene therapy can help prevent the disease from manifesting or progressing.
Overall, the primary objective of gene therapy is to cure genetic diseases by replacing or repairing defective genes with normal genes. This innovative approach holds great potential for the treatment and prevention of a wide range of genetic disorders, offering hope for improved health outcomes and quality of life for affected individuals.