Glossary - BioMarin HEMDifferently EN-US

Gene A part of a DNA molecule that tells your body how to make a protein. A mutation within certain genes can lead to lacking or missing proteins, which can lead to genetic conditions. Gene therapy aims to help the body produce the protein it needs.

Gene transfer therapy One method of gene therapy currently being explored in clinical trials. This approach aims to introduce a working gene into the body to evaluate whether it can produce a missing or deficient protein.

Genetics The study of genes and their heredity.

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