Gene therapy

Definition

Gene therapy is a technique that modifies or replaces faulty genes to treat or prevent diseases, often aiming to correct genetic disorders at their source. This innovative approach has significant implications across various fields, including medicine and biotechnology, as it offers potential solutions for previously untreatable conditions by targeting the underlying genetic causes.

5 Must Know Facts For Your Next Test

1. Gene therapy can be categorized into two main types: somatic gene therapy, which targets non-reproductive cells, and germline gene therapy, which alters reproductive cells and can affect future generations.
2. The first approved gene therapy in the United States was in 2017 for treating a rare genetic form of blindness called Leber's congenital amaurosis.
3. Viral vectors, such as lentiviruses and adenoviruses, are commonly used in gene therapy to facilitate the delivery of therapeutic genes into target cells.
4. Gene therapy has shown promise in treating various conditions, including inherited disorders, some types of cancer, and certain viral infections like HIV.
5. While gene therapy holds great potential, it also raises ethical concerns, particularly regarding germline modifications and the implications of altering human DNA.

Review Questions

• How does gene therapy utilize vectors to deliver therapeutic genes, and what are some common types of vectors used?
  • Gene therapy relies on vectors to transport therapeutic genes into target cells. These vectors can be viral or non-viral; viral vectors are often preferred due to their ability to efficiently infect host cells. Common types of viral vectors include adenoviruses, which can carry large DNA segments and have a broad range of host cells, and lentiviruses, known for their ability to integrate into the host genome, allowing for long-term expression of the therapeutic gene.
• Discuss the significance of somatic versus germline gene therapy and the ethical implications associated with each.
  • Somatic gene therapy targets non-reproductive cells and affects only the individual receiving treatment, while germline gene therapy involves modifications to reproductive cells that can be passed on to future generations. The ethical implications of germline therapy are profound, as they raise concerns about the long-term consequences of altering human DNA and the potential for unintended effects on descendants. Somatic therapies are generally considered more acceptable since their effects are limited to one individual, but both approaches require careful ethical consideration due to their potential impact on human health and evolution.
• Evaluate the advancements in gene therapy technologies like CRISPR and their potential impact on the future of genetic medicine.
  • Advancements in gene therapy technologies, particularly CRISPR, have revolutionized genetic medicine by enabling precise modifications of DNA sequences. This technology allows for targeted alterations that can effectively correct genetic mutations responsible for various diseases. The potential impact of CRISPR in gene therapy is enormous, as it not only enhances the efficiency of delivering therapeutic genes but also expands the range of treatable conditions. However, this power comes with ethical considerations regarding off-target effects and the long-term implications of making changes at the genetic level.

Related terms

CRISPR:

A revolutionary gene-editing technology that allows scientists to alter DNA sequences and modify gene function with high precision.

Vectors: Vehicles used to deliver therapeutic genes into a patient's cells; they can be viral or non-viral systems designed to efficiently transfer genetic material.

Monogenic disorders: Genetic conditions caused by a single defective gene, which are prime candidates for treatment through gene therapy.