Gene therapy

Definition

Gene therapy is a technique that modifies a person's genes to treat or prevent disease, often by correcting defective genes or introducing new ones. This approach has the potential to cure genetic disorders and certain types of cancer by targeting the root cause at a molecular level, and it heavily relies on techniques such as recombination and transposition to effectively deliver therapeutic genes into the patient's cells.

5 Must Know Facts For Your Next Test

1. Gene therapy can be classified into two main types: somatic gene therapy, which targets non-reproductive cells, and germline gene therapy, which affects reproductive cells and can be inherited by future generations.
2. One of the challenges of gene therapy is ensuring that the therapeutic gene is delivered effectively to the right cells without causing unwanted immune responses.
3. Recombination techniques are often used in gene therapy to integrate therapeutic genes into the patient's genome for stable expression.
4. Gene therapy has seen successful applications in treating conditions like spinal muscular atrophy and certain types of inherited blindness.
5. Regulatory considerations are critical in gene therapy, as ethical issues regarding germline modifications and potential long-term effects on patients must be carefully addressed.

Review Questions

• How does the use of vectors enhance the effectiveness of gene therapy?
  • Vectors are essential in gene therapy as they facilitate the delivery of therapeutic genes into target cells. By using modified viruses as vectors, scientists can harness the natural ability of these viruses to enter cells while ensuring safety through genetic modifications. This targeted delivery increases the likelihood that the therapeutic genes will be expressed properly within the patient's cells, making gene therapy more effective in treating various diseases.
• Discuss the role of CRISPR-Cas9 technology in advancing gene therapy techniques.
  • CRISPR-Cas9 technology plays a significant role in advancing gene therapy by providing a precise method for editing genes within the human genome. This system allows for specific changes to DNA sequences, enabling researchers to correct mutations that cause genetic disorders. The precision and efficiency of CRISPR-Cas9 make it a powerful tool for not only delivering genes but also ensuring that they are integrated correctly into the genome, thus enhancing the overall success rates of gene therapies.
• Evaluate the ethical considerations surrounding germline gene therapy and its implications for future generations.
  • Germline gene therapy raises significant ethical concerns because modifications made to reproductive cells can be passed on to future generations, leading to unforeseen consequences in the human gene pool. The potential for 'designer babies' brings forth debates about social equity, consent, and unforeseen health implications. These ethical dilemmas necessitate rigorous regulatory frameworks and societal discussions to ensure responsible use of this powerful technology while balancing innovation with moral responsibility.

Related terms

Vector: A vector is a vehicle used to transfer genetic material into cells, commonly derived from viruses that have been modified to be safe for therapeutic use.

CRISPR-Cas9: CRISPR-Cas9 is a groundbreaking gene-editing technology that allows scientists to make precise modifications to DNA, offering potential advancements in gene therapy.

Transduction: Transduction is the process by which foreign DNA is introduced into a cell through a viral vector, an essential step in many gene therapy strategies.