There are 2 main parts to gene replacement therapy: genes and vectors.
Check your understanding of gene replacement therapy
What does the new gene do?
Once the vector delivers the new gene to the cells, it then provides the instructions needed to make the protein that is missing or in short supply.
Can the viral vector make you sick?
While the vehicles used to carry the new gene into the nucleus of the cells are typically made from naturally occurring viruses, scientists have made changes to them so that they don’t make you sick. AAV, one type of virus used as a vector, is not known to cause sickness in humans.Learn more about how gene replacement therapy is different than other gene-based therapies.
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