Gene Therapy is Coming. Will it be Right for You?

If perfected, Gene therapy promises to heal in one shot, without complications or side effects. 


Imagine healing the body without drugs or surgery, each of which can have nasty side effects. Instead, a physician uses the body’s own building blocks to heal you. Instead of returning again and again, or having to take medication continually, one shot does it all. These are the promises of gene therapy.

The concept is easy to grasp. Genes control proteins that in turn control all of our body’s functions. When a faulty gene, usually due to a mutation, malfunctions and causes disease, all that would have to be done is to “knock out” or replace the faulty gene. Once the correct protein enters the system, the disease is finished. It is however the replacement process that is complicated.

One problem is exactly how to deliver a gene to a patient’s DNA. To do that, scientists create a custom virus that infects a target cell, yet flies under the immune system’s radar. By doing so, the virus leaves its own genetic material inside the cell. That cell begins to reproduce, carrying the gene with it, and spreading it throughout the body.

An adeno-associated virus (AAV) is used to deliver a gene to the DNA of a cell.

Gene therapies are not currently approved by the FDA. Dozens of clinical trials are ongoing, however. This cutting-edge therapy is approved to treat one particular disorder in Europe—lipoprotein lipase deficiency, where the patient cannot break down fat. Another use will soon be approved, to treat combined immune deficiency, or the “bubble boy” disease.

Other conditions it is expected to someday treat include heart disease, diabetes, some forms of cancer, muscular dystrophy, immune disorders, genetic disorders, AIDS, hemophilia, and certain blindness-causing conditions. With AIDS, gene therapy will be used in a different way. The HIV virus camouflages itself from the immune system. Gene therapy can make its presence known, allowing it to be recognized and destroyed.   

The things researchers look at when evaluating a new therapy is its safety profile, how effective it is, and what a proper dosage may look like. Just like any therapy, things can go wrong. For instance, altered viruses could change back into their original form, causing infection. Sometimes the wrong cell is approached by the virus. Or the virus places the gene in the wrong place within a cell’s DNA. In this last case, healthy cells may become damaged or cause illness, even develop into a tumor.


Model of gene therapy.

There have been stumbling blocks along the way. Keep in mind that all clinical trials are monitored by the FDA and the National Institutes of Health (NIH). Even so, gene therapy almost went bust in 1999 when a volunteer, 19-year-old Jesse Gelsinger, died during testing. The Arizona teen’s immune system reacted violently as a result of the treatment. Gene therapy lost its innocence and many young, promising scientists decided to put their efforts elsewhere, setting the field back. A year after that, during a French trial, some participants developed leukemia.

After these incidents, dozens of clinical trials ceased and funding was pulled. Researchers learned a lot from these disturbing tragedies, and put stringent safety controls in place. They have since discovered how to deliver genes using viruses in a safe and effective manner, that doesn’t set off the immune system.

Researchers have also implemented guidelines that help monitor patients and administer to side effects. A few successes then brought gene therapy back from the brink. In 2008, some blind subjects reported improvements in vision. Shortly after, in another experiment, 80% of “bubble boy” children regained immune system function.

Boy with combined immune deficiency or “bubble boy” disease.

The efficacy of gene therapy today is not constant throughout, but varies from one condition to the next. A recent study using the therapy to treat muscular dystrophy saw impressive results. A 2013 study was even more dramatic, where a small clutch of patients with leukemia were cured. Other studies on hemophilia and one cause of blindness, retinitis pigmentosa, have also seen remarkable results.

There have been other trials however that have not been so encouraging. One for congestive heart failure ended in “disappointing” results, and another for Parkinson’s ended in what researchers called a “mixed bag.” One problem that must be overcome, the immune system sometimes does recognize and take out the viral messenger.

Besides its checkered past and some obstacles to overcome, gene therapy does show promise for a lot of conditions. The biggest obstacle researchers foresee, it carries a hefty price tag. It costs one million dollars per treatment for the drug already approved in Europe. U.S. prices are expected to mirror this. Luckily, gene therapy is only applied once, and the effects are meant to last.

Down the road however, when this therapy becomes commonplace, it may actually save the healthcare system, as follow-up treatments will become superfluous. Born out of the 1980’s gene therapy, for at least one condition, may reach U.S. shores as soon as 2017, as an application filed with the FDA is set to be complete this year.

To learn more about gene therapy click here: 

3D Animation Gene Therapy.FLV from eLearn.Punjab on Vimeo.

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Political division is nothing new. Throughout American history there have been numerous flare ups in which the political arena was more than just tense but incideniary. In a letter addressed to William Hamilton in 1800, Thomas Jefferson once lamented about how an emotional fervor had swept over the populace in regards to a certain political issue at the time. It disturbed him greatly to see how these political issues seemed to seep into every area of life and even affect people's interpersonal relationships. At one point in the letter he states:

"I never considered a difference of opinion in politics, in religion, in philosophy, as cause for withdrawing from a friend."

Today, we Americans find ourselves in a similar situation, with our political environment even more splintered due to a number of factors. The advent of mass digital media, siloed identity-driven political groups, and a societal lack of understanding of basic discursive fundamentals all contribute to the problem.

Civil discourse has fallen to an all time low.

The question that the American populace needs to ask itself now is: how do we fix it?

Discursive fundamentals need to be taught to preserve free expression

In a 2017 Free Speech and Tolerance Survey by Cato, it was found that 71% of Americans believe that political correctness had silenced important discussions necessary to our society. Many have pointed to draconian university policies regarding political correctness as a contributing factor to this phenomenon.

It's a great irony that, colleges, once true bastions of free-speech, counterculture and progressiveness, have now devolved into reactionary tribal politics.

Many years ago, one could count on the fact that universities would be the first places where you could espouse and debate any controversial idea without consequence. The decline of staple subjects that deal with the wisdom of the ancients, historical reference points, and civic discourse could be to blame for this exaggerated partisanship boiling on campuses.

Young people seeking an education are given a disservice when fed biased ideology, even if such ideology is presented with the best of intentions. Politics are but one small sliver for society and the human condition at large. Universities would do well to instead teach the principles of healthy discourse and engagement across the ideological spectrum.

The fundamentals of logic, debate and the rich artistic heritage of western civilization need to be the central focus of an education. They help to create a well-rounded citizen that can deal with controversial political issues.

It has been found that in the abstract, college students generally support and endorse the first amendment, but there's a catch when it comes to actually practicing it. This was explored in a Gallup survey titled: Free Expression on Campus: What college students think about First amendment issues.

In their findings the authors state:

"The vast majority say free speech is important to democracy and favor an open learning environment that promotes the airing of a wide variety of ideas. However, the actions of some students in recent years — from milder actions such as claiming to be threatened by messages written in chalk promoting Trump's candidacy to the most extreme acts of engaging in violence to stop attempted speeches — raise issues of just how committed college students are to
upholding First Amendment ideals.

Most college students do not condone more aggressive actions to squelch speech, like violence and shouting down speakers, although there are some who do. However, students do support many policies or actions that place limits on speech, including free speech zones, speech codes and campus prohibitions on hate speech, suggesting that their commitment to free speech has limits. As one example, barely a majority think handing out literature on controversial issues is "always acceptable."

With this in mind, the problems seen on college campuses are also being seen on a whole through other pockets of society and regular everyday civic discourse. Look no further than the dreaded and cliche prospect of political discussion at Thanksgiving dinner.

Talking politics at Thanksgiving dinner

As a result of this increased tribalization of views, it's becoming increasingly more difficult to engage in polite conversation with people possessing opposing viewpoints. The authors of a recent Hidden Tribes study broke down the political "tribes" in which many find themselves in:

  • Progressive Activists: younger, highly engaged, secular, cosmopolitan, angry.
  • Traditional Liberals: older, retired, open to compromise, rational, cautious.
  • Passive Liberals: unhappy, insecure, distrustful, disillusioned.
  • Politically Disengaged: young, low income, distrustful, detached, patriotic, conspiratorial
  • Moderates: engaged, civic-minded, middle-of-the-road, pessimistic, Protestant.
  • Traditional Conservatives: religious, middle class, patriotic, moralistic.
  • Devoted Conservatives: white, retired, highly engaged, uncompromising,

Understanding these different viewpoints and the hidden tribes we may belong to will be essential in having conversations with those we disagree with. This might just come to a head when it's Thanksgiving and you have a mix of many different personalities, ages, and viewpoints.

It's interesting to note the authors found that:

"Tribe membership shows strong reliability in predicting views across different political topics."

You'll find that depending on what group you identify with, that nearly 100 percent of the time you'll believe in the same way the rest of your group constituents do.

Here are some statistics on differing viewpoints according to political party:

  • 51% of staunch liberals say it's "morally acceptable" to punch Nazis.
  • 53% of Republicans favor stripping U.S. citizenship from people who burn the American flag.
  • 51% of Democrats support a law that requires Americans use transgender people's preferred gender pronouns.
  • 65% of Republicans say NFL players should be fired if they refuse to stand for the anthem.
  • 58% of Democrats say employers should punish employees for offensive Facebook posts.
  • 47% of Republicans favor bans on building new mosques.

Understanding the fact that tribal membership indicates what you believe, can help you return to the fundamentals for proper political engagement

Here are some guidelines for civic discourse that might come in handy:

  • Avoid logical fallacies. Essentially at the core, a logical fallacy is anything that detracts from the debate and seeks to attack the person rather than the idea and stray from the topic at hand.
  • Practice inclusion and listen to who you're speaking to.
  • Have the idea that there is nothing out of bounds for inquiry or conversation once you get down to an even stronger or new perspective of whatever you were discussing.
  • Keep in mind the maxim of : Do not listen with the intent to reply. But with the intent to understand.
  • We're not trying to proselytize nor shout others down with our rhetoric, but come to understand one another again.
  • If we're tied too closely to some in-group we no longer become an individual but a clone of someone else's ideology.

Civic discourse in the divisive age

Debate and civic discourse is inherently messy. Add into the mix an ignorance of history, rabid politicization and debased political discourse, you can see that it will be very difficult in mending this discursive staple of a functional civilization.

There is still hope that this great divide can be mended, because it has to be. The Hidden Tribes authors at one point state:

"In the era of social media and partisan news outlets, America's differences have become
dangerously tribal, fueled by a culture of outrage and taking offense. For the combatants,
the other side can no longer be tolerated, and no price is too high to defeat them.
These tensions are poisoning personal relationships, consuming our politics and
putting our democracy in peril.

Once a country has become tribalized, debates about contested issues from
immigration and trade to economic management, climate change and national security,
become shaped by larger tribal identities. Policy debate gives way to tribal conflicts.
Polarization and tribalism are self-reinforcing and will likely continue to accelerate.
The work of rebuilding our fragmented society needs to start now. It extends from
re-connecting people across the lines of division in local communities all the way to
building a renewed sense of national identity: a bigger story of us."

We need to start teaching people how to approach subjects from less of an emotional or baseless educational bias or identity, especially in the event that the subject matter could be construed to be controversial or uncomfortable.

This will be the beginning of a new era of understanding, inclusion and the defeat of regressive philosophies that threaten the core of our nation and civilization.