Chapter 16
Treating Genetic Disorders and Using Genetics to Tailor Treatment
IN THIS CHAPTER
Delivering healthy genes to treat or cure disease
Discovering precision medicine
Examining the benefits of pharmacogenetics
The completion of the Human Genome Project (described in Chapter 8), along with the sequencing of nonhuman genomes, spawned an incredible revolution in the understanding of genetics. Simultaneously, geneticists raced to develop medicines to treat and cure diseases caused by genes gone awry.
Gene therapy, treatment that gets at the direct cause of genetic disorders, is sometimes touted as the magic bullet, the cure-all for inherited diseases and cancer. Gene therapy may even provide a way to block the genes of pathogens such as viruses, providing reliable treatments for illnesses that currently have none. Unfortunately, the shining promise of gene therapy has been hampered by a host of challenges, including finding the right way to supply the medicine to patients without causing worse problems than the ones being treated.
The advances in our understanding of genetics as it relates to disease and its treatment have also led to great advances in precision medicine. Precision ...
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