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Editorís focus: The gene genie is out of the bottle
June 2019
by Jeffrey Bouley  |  Email the author
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There’s a saying about how you can't put the genie back into the bottle. I mean, it really depends upon what version of the genie/jinn mythology you are reading. Is it Disney’s “Aladdin” or something darker? Kind of like the difference between the fairy tales we tell our children and the original versions that rival many horror films today.
 
But the fact is that ever since the Human Genome Project gained steam, we have been hurtling forward rapidly in the areas of genetics, genomics, gene therapies and more (just look at all the CRISPR headlines these days; it’s even the stuff of high school presentations now, based on what I saw at the open house visit to my daughter’s soon-to-be high school).
 
And, what it comes down to is this: The gene genie is out of the bottle (or lamp, depending on the story). And he/she isn’t going back in.
 
I’m fine with that, mostly, but I’m also reminded of the fact that the jinn (or genie, in Western parlance) could be a very dark and dangerous being. And so I have my concerns as we move forward. Will we exercise the wisdom and restraint we need to? Or will we go too far, too fast, without heeding the risks?
 
It was just last year that Chinese researcher He Jiankui shocked the scientific community by pretty much ignoring the widely held ethical standards and artificially conceiving genetically manipulated children in an effort to provide genetic protection from HIV infection. And now the word is out that the mutation he set out to introduce into human embryos using CRISPR may be linked to increased mortality. So not only did he do something most scientists currently consider unethical, he may have brought into the world children with shorter lifespans in an attempt to protect them from one disease.
 
Also very recently, we have renewed debate on the patenting of human genes. Certainly, editing this publication and writing for it gives me a great deal of understanding and even sympathy for the importance of patents to protect business and promote discoveries. But there are dangers in that, too.
 
In May, several U.S. senators jointly released draft legislation that would authorize patenting of laws of nature, products of nature and abstract ideas. The American Civil Liberties Union (ACLU), the Association for Molecular Pathology and more than 100 other organizations sent a letter to Capitol Hill expressing their opposition to the recent proposal, arguing it explicitly overrules Supreme Court precedent forbidding the patenting of human genes.
 
“Human genes are not a commodity that should be controlled by any one corporation or person,” wrote Kate Ruane, ACLU senior legislative counsel, in an official ACLU statement. “The Supreme Court unanimously ruled that companies cannot patent human genes. In [Association for Molecular Pathology v. Myriad Genetics, Inc.], the Supreme Court held that a business cannot patent a gene because it is a product of nature. Congress should not upend years of settled law to grant corporations exclusive rights to examine our genes and hinder much-needed genetic research, testing and treatments for a range of diseases.”
 
And yet another concern as our gene genie grants wishes (but perhaps not granting them the way we intended)? Cost.
Certainly, human genetic testing and sequencing have come down in price drastically (and quickly) since the days of the Human Genome Project and the initial years following its completion. And we can hope that gene therapies will follow a similar route.
 
But at the end of May, AveXis (a Novartis company) announced that the U.S. Food and Drug Administration has approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 gene. The treatment costs $2.1 million, making it the most expensive therapeutic compound around.
 
So, the gene genie is out. Let’s be careful how we phrase our wishes, shall we?

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