How Genome Sequencing Enables Precision Cell and Gene Therapies

Watch part 1 of the interview here.

In part 2 of this interview, Joyce Ohm, PhD, explains how the sequencing of the human genome has been foundational to the development of modern cell and gene therapies for cancer. She notes that cell therapies are typically designed to target a specific tumor antigen expressed on the surface of cancer cells. This kind of precision is only possible because genome sequencing allows researchers to identify the abnormal genetic changes that distinguish a tumor cell from a normal, healthy cell. Without the ability to sequence tumors and pinpoint these alterations, scientists would have no way to design therapies capable of selectively attacking cancerous cells while sparing normal tissue.

Ohm emphasizes that this principle extends beyond cell therapy to any genetically or epigenetically targeted treatment. Whether a therapy works by targeting a mutated protein, correcting a faulty gene pathway, or exploiting an epigenetic vulnerability, its development depends entirely on first being able to "see" what is abnormal in a given patient's tumor genome. In other words, the diagnostic step of sequencing is inseparable from the therapeutic step of treatment design—one cannot exist without the other.

She also draws an important distinction between two categories of mutations driving cancer. Some mutations are unique to an individual patient's tumor, arising sporadically during that person's lifetime (somatic mutations). Others are inherited, passed down through families across generations (germline mutations). Both types can serve as targets for therapy, but identifying which category a mutation falls into has implications for treatment strategy, prognosis, and even genetic counseling for family members.

Overall, Ohm's comments underscore a broader theme in oncology: the Human Genome Project and subsequent advances in sequencing technology didn't just expand scientific knowledge; they created the essential toolkit that makes precision medicine, including targeted cell and gene therapies, possible in the clinic today.

Read the full interview with Dr Ohm.