104k views
5 votes
although gene therapy presents hope that genetic disorders can be cured, has it been particularly effective so far?

User Parvat R
by
8.9k points

1 Answer

4 votes

Final answer:

Gene therapy has had some success in treating single-gene disorders by inserting normal genes to replace defective ones using viral vectors, but it remains an experimental technique with many challenges yet to be addressed.

Step-by-step explanation:

Gene therapy presents a promising approach to curing a variety of genetic disorders by inserting normal genes into cells that carry mutant genes. This technique has shown some success, particularly in diseases caused by single-gene defects such as cystic fibrosis, hemophilia, muscular dystrophy, and sickle cell anemia.

In this process, a correct version of the gene is delivered into human cells using a viral vector, often an adenovirus. Despite the potential, gene therapy remains largely experimental, with a few notable successes but still faces significant challenges and risks that need to be overcome before it can become a common treatment.

User Glerup
by
7.4k points