Gene Therapy Basics (2022 Update)

Summary

Gene therapy is a medical approach that targets the underlying causes of diseases by correcting or modifying genetic material. Most of our genetic information is stored in DNA within 23 pairs of chromosomes. Changes in this DNA can affect protein production, leading to various conditions. Gene therapy involves introducing new or modified genetic material, typically using viral vectors, to help cells produce the correct proteins. Two main methods, in-vivo and ex-vivo, deliver the therapy either directly into the body or by modifying the patient's cells outside the body before reintroduction. Though still facing challenges, gene therapy shows promise for treating genetic disorders.

Takeaways

• 🧬 Genetic information is stored in 23 pairs of chromosomes in our cells, made up of DNA, which determines our unique traits.
• 🧬 Specific sections of DNA called genes provide instructions for protein production, which plays a crucial role in how our bodies function.
• 🧬 Humans have between 20,000 and 25,000 genes, with two copies of each gene inherited from biological parents.
• 🧬 Minor changes in DNA within genes can alter how proteins work, affecting processes like breathing, walking, or digestion.
• 🧬 Genetic mutations can occur naturally or due to environmental factors, and while cells often repair these changes, some cause diseases.
• 💉 Gene therapy aims to treat or prevent diseases by addressing the root genetic cause, either by fixing or replacing faulty genes.
• 💉 Gene therapy involves delivering genetic material, DNA or RNA, into cells to modify protein production, either reducing harmful proteins or boosting beneficial ones.
• 🧪 Viruses are commonly used as vectors to deliver genetic material into cells, but they are modified to avoid causing disease.
• 🧪 Two main types of gene therapy exist: in vivo (direct delivery to the body) and ex vivo (modifying cells outside the body and reintroducing them).
• ⏳ Despite the promising progress, gene therapy still faces challenges, such as lengthy clinical trials and FDA approval processes.

Q & A

What is gene therapy?

Gene therapy is the use of genetic material to treat or prevent disease. It involves transferring genetic material, such as DNA or RNA, into cells to alter how proteins are produced by the body, aiming to fix or enhance cellular functions.

What role do chromosomes and DNA play in our bodies?

Chromosomes, made of DNA, store the genetic information that determines our unique traits. Humans have 23 pairs of chromosomes, and specific sections of DNA, called genes, carry instructions for making proteins that are essential for the body’s functions.

How do genetic variations occur?

Genetic variations can occur as cells age or are exposed to environmental factors like chemicals. These variations can also be inherited from parents. Cells often detect and repair these changes, but sometimes they lead to diseases or disorders.

How does gene therapy address the root cause of diseases?

Gene therapy addresses the root cause of diseases by correcting or replacing faulty genes. If genes are like blueprints for the body, gene therapy fills in missing parts or corrects errors in the blueprint, helping to treat or prevent diseases.

What is the difference between DNA and RNA?

DNA stores the full genetic blueprint of an organism, while RNA is a modified copy of that information, used by cells to create the right proteins. RNA translates the instructions stored in DNA into action.

What are the main ways gene therapy is delivered to cells?

Gene therapy can be delivered in two main ways: in vivo (inside the body), where the gene is injected directly into the person, or ex vivo (outside the body), where cells are removed from the body, modified with the gene, and then returned to the person.

Why are viruses used as vectors in gene therapy?

Viruses are used as vectors because they are naturally good at entering cells. In gene therapy, harmful parts of the virus are removed, and it is used to deliver therapeutic genetic material into cells without causing disease.

What safety measures are in place for using viral vectors in gene therapy?

All viral vectors used in gene therapy are tested extensively to ensure their safety. The disease-causing components of the virus are removed, and the vectors are checked in multiple tests before being used in humans.

What are some challenges in advancing gene therapy?

Challenges include the long process of testing potential therapies in clinical trials, getting FDA approval, and ensuring that the therapies are safe and effective. However, progress in gene therapy research is improving our ability to treat rare and debilitating diseases.

How might gene therapy impact the future of medicine?

Gene therapy has the potential to revolutionize medicine by treating and preventing diseases that were once considered incurable. As research advances, gene therapy could one day prevent the onset of diseases entirely, improving health outcomes for millions.