Gene Editing: Gold Nanoparticle Delivery Shows Promise

Juip Family with Dr. Francis CollinsAbout a month ago, I had the pleasure of welcoming the Juip (pronounced “Yipe”) family from Michigan to NIH. Although you’d never guess it from this photo, two of the Juip’s five children—9-year-old Claire and 11-year-old Jake (both to my left)—have a rare genetic disease called Friedreich’s ataxia (FA). This inherited condition causes progressive damage to their nervous systems and their hearts. No treatment currently exists for kids like Claire and Jake, yet this remarkable family has turned this serious health challenge into an opportunity to raise awareness about the need for biomedical research.

One thing that helps keep the Juips optimistic is the therapeutic potential of CRISPR/Cas9, an innovative gene editing system that may someday make it possible to correct the genetic mutations responsible for FA and many other conditions. So, I’m sure the Juips were among those encouraged by the recent news that NIH-funded researchers have developed a highly versatile approach to CRISPR/Cas9-based therapies. Instead of relying on viruses to carry the gene-editing system into cells, the new approach uses tiny particles of gold as the delivery system!

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NIH Family Members Giving Back: Rebecca Shlafer

Rebecca Shlafer

Rebecca Shlafer/Credit: Brady Willette

When Rebecca Shlafer clicks on her office lights each morning at the University of Minnesota Medical Center, Minneapolis, she usually has a good idea of what to expect from the day ahead as lead of a nine-person research team that studies the effects of incarceration on children and families. It’s her volunteer work that can be unpredictable.

For the past eight years, this developmental child psychologist has donated her free time to serve as a guardian ad litem for abused or neglected children who’ve been removed from their homes and placed under protective supervision of Minnesota’s Fourth Judicial District. In that volunteer capacity, Shlafer advocates in court for the well-being of the child, but doesn’t foster the youngster or provide any day-to-day care.

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All Scientific Hands on Deck to End the Opioid Crisis

Word cloudIn 2015, 2 million people had a prescription opioid-use disorder and 591,000 suffered from a heroin-use disorder; prescription drug misuse alone cost the nation $78.5 billion in healthcare, law enforcement, and lost productivity. But while the scope of the crisis is staggering, it is not hopeless.

We understand opioid addiction better than many other drug use disorders; there are effective strategies that can be implemented right now to save lives and to prevent and treat opioid addiction. At the National Rx Drug Abuse and Heroin Summit in Atlanta last April, lawmakers and representatives from health care, law enforcement, and many private stakeholders from across the nation affirmed a strong commitment to end the crisis.

Research will be a critical component of achieving this goal. Today in the New England Journal of Medicine, we laid out a plan to accelerate research in three crucial areas: overdose reversal, addiction treatment, and pain management [1].

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Moving Toward Answers in ME/CFS

Woman in bed

Thinkstock/Katarzyna Bialasiewicz

Updated September 27, 2017: The National Institutes of Health (NIH) will award four grants to establish a coordinated scientific research effort on myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). The total cost of the projects for fiscal year 2017 will be over $7 million, with support from multiple NIH Institutes and Centers that are part of the Trans-NIH ME/CFS Working Group.

The grants will support the creation of a consortium made up of three Collaborative Research Centers (CRC) and a Data Management Coordinating Center (DMCC). The CRCs will each conduct independent research but will also collaborate on several projects, forming a network to help advance knowledge on ME/CFS. The data will be managed by the DMCC and will be shared among researchers within the CRCs and more broadly with the research community.


Imagine going to work or school every day, working out at the gym, spending time with family and friends—basically, living your life in a full and vigorous way. Then one day, you wake up, feeling sick. A bad cold maybe, or perhaps the flu. A few days pass, and you think it should be over—but it’s not, you still feel achy and exhausted. Now imagine that you never get better— plagued by unrelenting fatigue not relieved by sleep. Any exertion just makes you worse. You are forced to leave your job or school and are unable to participate in any of your favorite activities; some days you can’t even get out of bed. The worst part is that your doctors don’t know what is wrong and nothing seems to help.

Unfortunately, this is not fiction, but reality for at least a million Americans—who suffer from a condition that carries the unwieldy name of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), a perplexing disease that biomedical research desperately needs to unravel [1]. Very little is currently known about what causes ME/CFS or its biological basis [2]. Among the many possibilities that need to be explored are problems in cellular metabolism and changes in the immune system.

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Is Social Media Making Us Lonelier?

Social mediaInitially, most of us thought that Facebook, Instagram, Twitter, and other social media applications would help to bring people together. And, yes, in many instances that has been true. Such apps have made it possible—even simple—to catch up with former classmates living thousands of miles away, share a video of your baby’s first steps with relatives near and far, or strike up new acquaintances while discussing the stock market or last night’s ballgame. Yet, a new NIH-funded study suggests that social media may also have the power to make people feel left out and alone.

Based on a nationwide survey of more than 1,700 young adults, researchers found that individuals who were the heaviest users of social media were two to three times more likely to feel socially isolated than those who used little to no social media [1]. And that’s a concern to those of us in the medical field: previous research has linked social isolation to worsening physical and mental health, and even an increased risk of death [2,3]. In fact, some experts have gone so far as to label loneliness a major public health concern.

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