What is the goal of gene therapy?

What is the goal of gene therapy?

• A. To treat or prevent disease by correcting defective genes or delivering therapeutic genes
• B. To clone organisms
• C. To sequence genomes faster
• D. To enhance athletic performance

Answer: (A)

The goal of gene therapy is to treat or prevent disease by correcting defective genes or delivering therapeutic genes to cells. This means repairing a faulty mutation so the cell can make the right protein again, replacing a malfunctioning gene with a working copy, or editing the genome to disable a harmful variant. Techniques often involve delivering a healthy gene using a vector or using gene-editing tools to make precise changes at the disease site. The emphasis is on medical benefit for genetic disorders, not on cloning organisms, sequencing DNA, or enhancing performance. Cloning creates genetic copies, sequencing reads and interprets DNA, and enhancement aims at improvements rather than treating disease.

The goal of gene therapy is to treat or prevent disease by correcting defective genes or delivering therapeutic genes to cells. This means repairing a faulty mutation so the cell can make the right protein again, replacing a malfunctioning gene with a working copy, or editing the genome to disable a harmful variant. Techniques often involve delivering a healthy gene using a vector or using gene-editing tools to make precise changes at the disease site. The emphasis is on medical benefit for genetic disorders, not on cloning organisms, sequencing DNA, or enhancing performance. Cloning creates genetic copies, sequencing reads and interprets DNA, and enhancement aims at improvements rather than treating disease.