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Genetic advances bring great possibilities, but also require responsibility

Published Sept. 21, 2016

In January 2009, I wrote a guest column in the Tampa Tribune titled "Unraveling Genome Has Great Potential, But We Are Not There Quite Yet."

Little did I know then that, by 2015, we would be there!

We have known for a long time that in humans, life is transmitted through 23 pairs of chromosomes in each cell, half of each pair contributed by each parent. The chromosomes are made of DNA, which is encoded into various genes.

In 1953, James Watson and Francis Crick unveiled the structure of DNA, cracking the code of life. Decades later, the entire human genome (all of the DNA sequences in a single cell) was mapped, made of billions of nucleotides (the chemical alphabet). The scientists then focused on identifying the tiny portions of the gene that are responsible for various functions and dysfunctions.

For a while, scientists toyed with the idea of altering DNA (the basic units of the gene), finally resulting in genetic engineering with recombinant DNA (cut and paste — cut the nucleotides from the genes of one organism and paste them into the genes of another) to introduce desired traits.

For example, by altering the genes of bacteria, large quantities of hormones (insulin, for instance), antibiotics and clot-busting medications were manufactured for human medical treatment, saving millions of lives. Similar benefits were achieved from transgenic animals.

For the past couple of decades, the technique was also used to produce transgenic crops called GMOs (genetically modified organisms) by splicing genes from one species into a different species to improve productivity and to enrich their quality.

For the past few years, scientists have embarked on the idea of "gene editing," a process that nature does all by itself to protect bacteria from viruses. The scientists observed it, learned from it and duplicated it in animals and humans.

When a bacterium is invaded by a virus, it keeps a genetic record of the virus in memory and when re-invaded by the same virus, it produces a powerful enzyme that effectively snips the virus out (a molecular scissor). Scientists named the process "CRISPER" and the enzyme "Cas9."

This process of gene editing, which can be done on any living organism, has enormous potential in infinite ways to improve on nature. The applications can range from cancer research to curing diseases, production of vaccines to eliminating mosquito-born illnesses, production of fuel and electricity to disposal of plastic and production of super crops to save endangered species.

This new genome-editing technique (deleting, altering or rearranging DNA) is precise, relatively quick and easy, and inexpensive. A CRISPER kit can be commercially bought for $130.

The story is all rosy, until one thinks of using this technique to edit germ-line cells (sperm, egg, embryo). The resulting change can be passed on to future generations forever. What might be the long-term unintended consequences of altering the genes permanently? A rogue scientist could go beyond the ethical, moral and legal limitations, producing designer babies, superior humans and super bugs.

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Are we trying to play God? I wouldn't even venture to go there in this column. However, I cannot help but wonder why the creator is empowering humans with super-intelligence to decode all the secrets of creation and the means to alter it in no small measure. What is the creator's ultimate plan? Only God knows.

But the genie is out of the bottle, and gene editing is here to stay. This technology is one of the biggest things since nuclear energy. We can only hope that it will be put to good use.

Dr. Rao Musunuru is a practicing cardiologist in Pasco County and a past member of the advisory council for National Heart, Lung and Blood Institute at the National Institutes of Health. His son, Kiran, also a cardiologist, is at the forefront of the genomic revolution and recently received a presidential award.