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What does "Gene Therapies" mean?

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Gene therapies are medical treatments that aim to fix or replace faulty genes in order to treat diseases. They are often used for conditions caused by genetic problems, which can be very serious and life-threatening.

How Gene Therapies Work

Gene therapies work by delivering healthy genes into a patient's cells. This can help the body make the right proteins it needs to function properly. Sometimes, gene therapies can also help the body stop making harmful proteins that cause disease.

Types of Gene Therapies

There are different types of gene therapies, including:

  1. Replacing Missing Genes: Some diseases happen because a gene is missing. Gene therapy can replace that missing gene.
  2. Fixing Faulty Genes: If a gene is present but not working right, gene therapy can help correct it.
  3. Adding New Genes: In some cases, doctors can add new genes to help the body fight a disease.

Challenges in Gene Therapies

While gene therapies show great promise, there are challenges. Understanding how well these treatments work over the long term can be difficult, especially when data is limited. Researchers are looking for ways to gather more information to better predict the outcomes of these therapies.

Future of Gene Therapies

Researchers continue to study gene therapies to improve their effectiveness and safety. This includes finding better ways to deliver the genes and ensuring that they work properly in the body. As new techniques develop, gene therapies may become a valuable option for treating a wider range of diseases.

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