gene replacement therapy could help change lives
What is gene replacement therapy?
Gene replacement therapy is a type of gene-based therapy that works to correct an underlying genetic condition at its root cause, the gene.
Learn about different types of gene-based therapiesWhat research is being done in genetic diseases?
Genetic disease research is making important advancements in our understanding of many genetic diseases and how they can be treated, including cystic fibrosis, hemophilia, and spinal muscular atrophy. Today, more than 2,600 gene therapy clinical trials are ongoing or have been completed worldwide.
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