Gene Therapy – The Fix Is (Almost) In

Since its completion in 2003, the Human Genome Project has garnered a great deal of attention for genetic testing. All of this focus has resulted in the identification and mapping of the three-billion pairs of human gene structure, known as the genome. Today, geneticists can perform many different types of analyses that have never been seen before, including deep sequencing, RNA sequencing and epigenomics, the overall study of the complete set of epigenetic modifications on the genetic material of a cell – all of these allow scientists to study the expression of genetic patterns from person-to-person and more.

Genetic therapy is a developing area of our unprecedented understanding of the human genome. Imagine being able to add a normal, healthy gene where a person has inherited a defective gene variant? In some cases, this may improve outcomes from a disease and in the best cases, cure or prevent it altogether.

One of the good news stories coming out of 2020, is the work being advanced by biochemists Jennifer Doudna and Emmanuelle Charpentier – first-time ever, female co-winners of the Nobel Prize in chemistry. These brilliant scientists have developed a method for genome editing called Clustered Regularly Interspaced Palindromic Repeats, also known as CRISPR – a type of genome editing that provides a way to cut into our DNA code, making precise alterations to our genetic make-up, one that might change the very course of medical therapy (1).

CRISPR technology has already made a difference to at least one person. Victoria Gray, an American in her 30s, was born with sickle cell disease (SCD) – an illness related to a defect in a single gene that can result in many complications, has overall reduced survival and when it comes to insurance coverage, is not generally an insurable risk. Until now, treatments have included medications that are not curative, although in some cases, stem-cell transplants have been successful (2).

Ms. Gray underwent CRISPR therapy in 2019, receiving billions of genetically modified cells via infusion to correct her SCD. More than a year later, she continues to thrive with no further signs of the disease (3). CRISPR therapy provides a great deal of hope for other types of diseases such as cancer and other widespread illnesses. Much like the discovery of insulin in the 1920s by the Canadian icon Dr. Fred Banting which led the way to the improved survival (and insurability) of millions of diabetics, this new decade (albeit a challenging start) may offer new knowledge and improved health. We are certainly not there yet… but there is hope on the horizon.

Widely available gene therapy holds much promise in literally changing the course of an individual’s life, improving both the quality of life and increasing overall life expectancy. At that time, the impact on underwriting – insuring more lives at more competitive rates – will be significant.

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  1. The Nobel Prize. Press release: The Nobel Prize in Chemistry 2020. nobelprize.org. October 7, 2020.
  2. National Library of Medicine. Sickle cell disease. PubMed.gov. March 15, 2018.
  3. Stein, Rob. The CRISPR Revolution. NPR.org. June 23, 2020.

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Gene Therapy – The Fix Is (Almost) In