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Genomic Medicine: Unique Considerations for Rare Disease Clinical Trials

Genomic medicine is an emerging field of study that looks at one’s genes to determine how negative changes in an individual’s DNA may result in disease and uses that knowledge to create a treatment to correct the error. Gene therapy, genome editing, gene-edited cell therapy and genome regulation are all…

A Closer Look at Gene Therapy

Our genes make us unique. Inherited from our parents, genes help determine physical features and traits like height, eye and hair color. Genes are made up of DNA, which are genetic instructions to build enzymes and proteins. Proteins make muscles, bones, and blood, among many other things in the body.