How Controversial Gene Editing Could Lead To Groundbreaking Cures

Thanks to the controversial new technology known as CRISPR, scientists are beginning to make headway in understanding and potentially curing some of the world's most intractable diseases.

Sickle-cell anemia, HIV, schizophrenia and autism -- essentially, anything involving bad DNA is now fair game. The latest example, from a study published earlier this month in the journalMolecular Therapy, focuses on Facioscapulohumeral muscular dystrophy, or FSHD, which is one of the most common forms of muscular dystrophy. The genetic disease causes the muscle fibers in the face, shoulders and upper arms to weaken over time -- and there is no known cure.

Enter CRISPR. This new gene-editing technique allows researchers to easily change, delete or replace genes in any plant or animal, including people. Picture the precision and ease of the find-and-replace function on a word document -- that’s how easy it now is to change the human genome. As an article in the MIT Technology Review put it last year, “This means they can rewrite the human genome at will.” Or, as one bioethicist told The Huffington Post last week, comparing what CRISPR can do to earlier attempts at genetic manipulation, “We used to have a butter knife, now we’ve got a scalpel.”

Biomedical researchers all over the world are now wondering how the technology might change their approach to all sorts of diseases. About a year ago, a team of FSHD researchers, led by Peter Jones at the University of Massachusetts Medical School, decided to give CRISPR a try. They already had a pretty good idea which of the thousands of genes in the human genome caused the disease, but until CRISPR came along therapeutic avenues were limited.

The acronym CRISPR, which stands for (take a deep breath) “clusters of regularly interspaced short palindromic repeats,” refers to both a technique and an actual thing, which is a macromolecular complex. The complex consists of a mix of two different types of biological material, protein and RNA. The RNA seeks out and binds to the targeted gene, like a hunting dog chasing down a fox, and the protein goes to work on it -- the hunter firing a shot.

Why would someone want to hunt down a gene? There’s no one answer to that question. Bad genes are like Tolstoy’s unhappy families -- they’re all bad in different ways. Sometimes a mutated gene doesn’t make the protein it’s supposed to make. When that’s the case, CRISPR can be used to replace this lazy gene with one that actually does its job.

Other times, the gene isn’t lazy, if you will, but is actively malicious -- it makes a protein that it’s not supposed to make, a toxin. That’s the story with FSHD. The U. Mass team figured they’d try to use CRISPR to shut down the gene, to “turn it off.” Until they got started on this study, no one had ever proved that CRISPR could be used to turn off a human disease gene, and the U. Mass researchers themselves weren’t sure it would work.

Well, it did.

The implications of this are pretty huge. The research could pave the way for other valuable studies, according to Charis Himeda, the lead author on the study. “I think progress for any disease is really progress for all diseases, because a lot of these therapies and technologies are going to turn out to be broadly applicable,” she said.

 It’s important, however, not to get carried away. Himeda said she didn’t know specifically which other diseases could possibly be cured as a result of CRISPR inhibition, and she thinks it’s too soon to start testing any of the CRISPR techniques on people. ‘I think there’s reason to be really hopeful that some day it’ll actually lead to great therapies for genetic diseases as long as we’re not too eager to get this to the clinic right away,” she said.

Himeda’s use of CRISPR may be less risky than some of the other potential therapies. Replacing bad genes, as other scientists have done, involves cutting away pieces of DNA. To simply stop them from making stuff, as the U. Mass team has done, is less likely to cause the genes any permanent damage. But that doesn’t mean the technique is risk-free. Himeda points out that while they achieved a 50 percent reduction in expression of the FSHD disease gene, we still don’t know what the effects are on all the other genes in a cell. “There are things that we don’t understand yet that we need to characterize before we move forward,” Himeda said. 

There’s a bigger ethical question at play here, too. Many people think it’s simply a bad idea to meddle with the human genome, no matter what technique you use or how good your intentions are. The fact that CRISPR is so easy to use makes it especially frightening. What happens if it ends up in the wrong hands? If sci-fi has taught us anything, it’s that it won’t end well. Think the 1997 futuristic thriller "Gattaca," where some children are conceived through genetic engineering to ensure a perfect -- and disease-free -- life, while others, known as "in-valids," are relegated to a brutal existence of menial labor.

Even Himeda worries about that. “People talk about it as a great tool, and it’s promising, but it’s also scary,” she noted. “We’re not ready for ‘Gattaca.’”

Related coverage:

* This Technology Can Fight Cancer And Create Adorable Mini Pigs. So Why Are Scientists So Worried?

Science Has Determined How Not To Look Stupid

Whether it's your sibling, best friend, parent or even your boss, you've called someone stupid at some point in your life. But how do we even define "stupid" behavior, and what can we do to avoid acting stupidly?

A new study offers a scientific answer.

"Although calling something stupid is a frequent everyday behavior," Balázs Aczél, apsychology professor at Eotvos Lorand University in Budapest and a co-author of the study, told The Huffington Post in an email, "there has been no psychological study to understand why and when people use this label to describe the observed actions."

Aczél and his colleagues gathered 180 pieces of writing from the news, blog sites, and social media that might be deemed as "stupid." The pieces were then presented to 154 adults who were asked to fill out a survey about each story.

The study participants answered whether they thought the behaviors and actions in the writing clips were stupid, and how stupid they considered them to be on a scale from one to 10.

The researchers found that, despite the ambiguous definition of "stupid," there was a 90 percent rate of agreement between participants in which actions were seen as stupid. The researchers also noticed that there were three different types of behaviors, which were most often deemed as stupid.

"In our statistical analysis of the data we found that people regard stupid action in three different categories: (1) violations of maintaining a balance between confidence and abilities; (2) failures of attention; and (3) lack of control," Aczél said.

Here are examples of the three categories:

1. Overconfidence 

Aczél described it as "confident ignorance," or when someone is overconfident about their ability to do something. An example of this behavior is when a driver refuses to ask for directions, and might end up lost. 

"What that tells us is that you don’t have to have a low IQ, in people’s eyes, to act stupidly. You just have to misperceive your abilities,"Aczél told The Washington Post. 

2. Lack of control

A lack of willpower or control might be when your friend is on a diet but buys cookies in the grocery store "just in case."

3. Absent-mindedness 

Absent-minded behavior could be described as when someone who buys a car doesn't know how to change the oil, and thus, the vehicle breaks down on the side of the road.

The researchers noted that more research is needed to determine how much of their findings are influenced by culture and shared expectations.

But for now, "these results bring us closer to understanding the rationalistic norms that people use in monitoring and evaluating behavior," they concluded.

The study was published in the November-December 2015 edition of the journal Intelligence.

HELPING KIDS DEAL WITH THEIR FEARS, PART II Janel Breitenstein Mom Since 2004 Editor’s note: Last week, Janel wrote about a situation with her daughter and how she and her husband dealt with her fears. Click here to read part one, and read on to see the rest of Janel’s tips. Help your child develop healthy coping mechanisms. This is where Scripture memory comes in. Perhaps they also take deep breaths, visualize themselves confronting the fear with confidence, etc. For my son with ADHD-related anxiety, I laminated a pocket-sized card that listed strategies for when he felt out of control: “I can find a place to be alone. I can find one thing to be thankful for…” Sometimes you’ll be choosing whether or not to completely remove your child from a fearful situation or allow him or her the important teaching experience—and emotional muscle—of learning from hard stuff as you talk each encounter out. My parents kept me in the classes of at least two teachers I hated, and it changed me in very good ways. One taught me how to write well, which is now my career. And their resolve (face it—it’s hard to watch our kids suffer!) helped me to keep my 4-year-old on the team of a bad soccer coach, so he could learn to deal with more difficult personalities, and we could talk it out in the car after I kept an eye from the sidelines. Be careful not to baby kids’ fears—especially those that someday, they will have to face on their own (Water. Dogs. New people.). Avoiding scary situations doesn’t help our children develop scaffolding to conquer them. And God has a lot of great promises for overcomers and conquerors! Take the next step. What steps can your child use to address the practicalities of his fear? What would the first, itty-bitty step be? This site suggests using yourself as a “home base” to which the child can repeatedly return after approaching the feared object. If your son or daughter continually struggles with fear, consider other ways you can increase confidence. My husband gained a great deal of confidence in his teen years through hiking, mountaineering, kayaking, and other physical challenges with his dad. Other children develop poise, fluidity, and perseverance through sports, or in developing one of their unique areas of talent—like performing arts, for example—that require “little bites” of confidence and courage. Maybe that means praising your son as he tries bigger stunts at the playground, or your daughter as she refutes peer pressure or decides not to back down in the face of more popular girls at school. For some kids, realize that talking too much about the issue cements them further in their fear. Yep, I would agree that there are times when our kids just need to choose courage: We’ve talked it out, we’ve prayed about it, and now it’s just time to choose the right thoughts, and not choose others. Getting them to laugh a little might help! Obviously navel-gazing is not biblical, either—and in our self-focused world, sometimes we can take attending to our kids’ emotions to the extreme of self-focus, and perpetuate fear. Neuropsychology teaches us that once the brain explores a new concept, it’s much easier to revisit that concept; our brain has already breached pathways there. For me, this is another great reason to make every thought obedient to Jesus Christ. Our words, and even our thoughts, have power in cementing what we think. Reward them for courage—even if it’s just verbally. If your son braved the dentist, perhaps he could watch a short DVD when you get home. Create positive memories, feelings, and reinforcement to surround acts of courage. My daughter’s “baby step” to her friend’s home was a good first step to confronting her fears. And when we picked her up giggling later that night, it was good to gently remind her that her fears were not realized, tell her we were proud of her, and snuggle her up in our arms. Janel has recently launched a new blog of her own: A Generous Grace. She writes, “Discovering God’s undeserved, indescribable favor—grace—has turned my life on its head. It’s my hope that He’ll knead that more into my soul, so others can soak it up, too. This site’s about ‘Jesus with jeans on’—letting God work Himself into real, practical, everday life.” Check it out!