Gene Therapy: Chinese Researching Gene Replacement

When I mention the words gene therapy, you might immediately think of the horrifying eugenics experiments conducted by the Nazis in World War II.

Since then, however, fantastic advances in technology have allowed scientists to learn an incredible amount about how the human body works. As a result, gene therapy and genetic medicine have become a huge boon to mankind since the practice originally emerged.

Indeed, initiatives like the Human Genome Project have shed more light on genetic expression and how various traits and diseases are passed from generation to generation.

In fact, gene therapy currently in its infancy holds the promise of suppressing (or even curing) any number of genetic diseases or disorders.

And in this regard, scientists in China have just taken a bold new step forward. One that others have feared to take

Along with the very first thought about gene therapy came a huge concern: The very same therapies that could modify human genes in existing patients could be used to modify people before they were even born.

You see, when gene therapy is conducted on fully formed, living people, the results arent heritable. So if something goes wrong, its limited to that person any man-made genetic mistake wouldnt spread into the wider population.

But a genetically modified embryo could theoretically carry the new genetic information through every cell it ever grew including reproductive cells and pass the modification on to his or her children.

This fear, not to mention the specter of less ethical gene modifications in the future, has previously blocked scientists from performing gene replacement experiments on human embryos.

But not anymore

Scientists at Sun Yat-sen University in Guangzhou, China recently took an unprecedented step.

They replaced the genes in a single-cell embryo.

Their motives were well-intentioned the experiment was to try to replace a gene that causes a serious and sometimes fatal blood disorder, and which replicated similar gene replacement therapies tested on adult human cells and animal embryos.

They also took some precautions to allay ethical concerns. Lead scientist, Junjiu Huang, insisted on using non-viable embryos i.e., castoffs from fertility clinics that could never become fully developed humans.

Nevertheless, some scientists are up in arms about the experiment, despite the precautions taken. And despite the significance of the research, it caused enough controversy that the resulting report was said to have been rejected by some larger scientific journals.

Unfortunately, the experiment didnt produce the expected results anyway. The existing gene-replacement regimen didnt result in successful gene replacement often enough to justify any further experimentation along the same line.

But still, the step has been taken and its an important one

Ultimately, were nearing a day when it will be possible to cure diseases and disorders before people get them even before theyre born! Thats obviously a goal worth pursuing. And while some scientists dislike gene experimentation, others are more philosophical, reasoning that its only a matter of time before such trials are commonplace.

But were not there yet.

Gene replacement therapy is still a young science, and despite hundreds of therapies that have gone to human clinical trials, there isnt a single gene replacement therapy in widespread use.

Indeed, one of the more successful gene replacement treatments was actually stopped. Scientists found that while it did cure the disease it targeted, the new genetic code also seemed to cause leukemia.

Thats a prime example of how complex this field is and the danger of using such therapy on embryos. An unintended disease or disorder could be passed down from parent to child but perhaps not becoming apparent for many generations and long after that code is widespread.

However, with the experiment at Sun Yat-sen University having been completed, its clear that we no longer have the luxury of deferring the argument about the ethics of genetic changes to human embryos to some point in the future. Because the future has just occurred.

To living and investing in the future,

Greg Miller

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Gene Therapy: Chinese Researching Gene Replacement

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