Now, we have a potential therapeutic target to accomplish this: a family of microRNAs called miR-17-92 that regulates cardiomyocyte proliferation. In Circulation Research earlier this month, a team led by Kühn’s research colleague Da-Zhi Wang, PhD, demonstrates its potential. Full story »
From the category archives:
As a hematologist, I see all too many children battling blood disorders that are essentially untreatable. Babies with immune deficiencies living life in a virtual bubble, hospitalized again and again for infections their bodies can’t fight. Children disabled by strokes caused by sickle cell disease, or suffering through sickle cell crises that drug treatments can’t completely prevent. Children whose only recourse is to risk a bone marrow transplant—if a suitably matched donor can even be found.
Over the past 20 years, my lab and that of George Daley, MD, PhD, at Boston Children’s Hospital have worked hard to give these children a one-time, potentially curative option—a treatment that begins with patients’ own cells and doesn’t require finding a match. Full story »
Thalidomide has its own fascinating history. Originally developed by Chemie Grünenthal GmbH in the 1950s, it was the result of a search for an anti-anxiety drug to compete with Valium, and was approved for use in Europe as a sleep aid and depression treatment. Eventually, doctors found it useful for treating nausea, and started prescribing it off-label to pregnant women with morning sickness.
The results were disastrous. Thalidomide turned out to be a teratogen, causing severe birth defects. Full story »
Lindsay Hoshaw contributed to this post.
It’s 7 a.m. and neurosurgeon
Ed Smith, MD, is downing a Diet Coke as he reviews the MRIs of today’s patients. He sprints up a stairwell to greet his first patient in the pre-operating wing.
Thirteen-year-old Maribel Ramos, about to have brain surgery at Boston Children’s Hospital, sits in her bed fidgeting. Smith reassures her about the operation, promises they’ll shave off as little hair as possible, and gets Maribel to crack a smile by telling her he moonlights as a hairdresser. Full story »
A technology from a small research institute, originally developed as a safer way to make embryonic-like stem cells, just hooked a very large fish. As The New York Times reported yesterday, pharma giant AstraZeneca is betting at least $240 million that this technology could be the source of a variety of new drugs—drugs that spur the body itself to make what it needs.
In 2010, the lab of Derrick Rossi at the Immune Disease Institute, which is now the Program in Cellular and Molecular Medicine at Boston Children’s Hospital, reported that they could reprogram ordinary cells into pluripotent stem cells by simply injecting them with messenger RNAs. The mRNAs reprogrammed the cells up to 100 percent more efficiently than other techniques, and did so without becoming part of the cell’s genome, greatly reducing concerns about cancer associated with other methods.
Key to the discovery were the chemical modifications made to the mRNAs so that cells wouldn’t “see” them as viruses and attack them. This video and this article describe the modified mRNA technique, also described in Cell Stem Cell:
I recently took my 6-year-old son to a Family Science Day, hosted by the 2013 American Association for the Advancement of Science (AAAS) Annual Meeting in Boston. He was most excited by a model airplane made out of parts that had been generated with a 3D printer. The scientist, from MIT, explained to us how this technology works: Instead of generating 2D printouts by spraying ink onto paper, 3D printing technologies assemble 3D objects layer by layer from a digital model, generally using molten plastics or metals.
3D printing is quickly being adopted by many professions, from architects and jewelers who want to build mock-ups for clients, to manufacturers of products like bikes, cars or airplanes. Soon we might all have 3D printers in our homes: The kids could design and print their own toys, while the grownups might use the technology to generate replacement parts for minor home improvement jobs (our broken shower faucet knob comes to mind). Full story »
There’s a widespread view that attention-deficit hyperactivity disorder (ADHD) is grossly over-treated in kids, especially boys, and will eventually be outgrown. But the results of the first large, long-term population-based study, published recently in Pediatrics, suggest that couldn’t be further from the truth.
While other studies have indicated dire outcomes when children with ADHD grow up, most of these have been small and have focused on the severe end of the spectrum—for instance, boys referred to psychiatric treatment facilities. This new study, started at the Mayo Clinic and led by William Barbaresi, MD, looked at the general population of kids with ADHD and found a greater likelihood of their having other psychiatric disorders as adults, doing jail time or committing suicide.
“Only 37.5 percent of the children we contacted as adults were free of these really worrisome outcomes,” says Barbaresi, now at Boston Children’s Hospital. “That’s a sobering statistic that speaks to the need to greatly improve the long-term treatment of children with ADHD and provide a mechanism for treating them as adults.” Full story »
With the Internet’s meteoric rise in the last 20 years—to the point of being available 24/7 in your pocket—technology pundits, psychologists and sociologists have been sounding ever louder warnings about information overload: the constant onslaught of communication, information and media coming at us all the time, and in ever greater volume.
Now imagine you’re a doctor or nurse in an intensive care unit (ICU). For you, information overload isn’t just a daily reality—it’s a necessary one. To make the right decisions at the right time for each patient, you must keep tabs on numerous bedside monitors—in the ICUs at Boston Children’s Hospital, that’s 10 or more for each child.
Melvin C. Almodovar, MD, medical director of Boston Children’s Cardiac Intensive Care Unit (CICU), and his colleagues wanted a better way to assess the patient’s physiologic state and catch crises before they happen. Full story »
Right now, immunizations against most infections begin at 2 months of age. But that leaves newborns at risk for infections like rotavirus, whooping cough and pneumococcus during a highly vulnerable time.
In resource-poor countries, this is a serious problem: Many children see a health care provider only at birth, so may miss their chance to be protected. Worldwide, each year, more than 2 million infants under 6 months old die from infections, especially pneumonia. If we could immunize infants at birth, it would be a huge win for global health.
Unfortunately, though, newborns don’t respond to most vaccines. Their immune systems are too immature—which is why few vaccines for newborns exist. Full story »
Ed note: The Obama administration is expected to unveil plans for a decade-long Brain Activity Map project next month. This is Part One of a two-part series on brain mapping.
It’s now pretty well accepted that autism is a disorder of brain connectivity—demonstrated visually with advanced MRI techniques that can track the paths of nerve fibers. Recent exciting work analyzing EEG recordings supports the idea of altered connectivity, while suggesting the possibility of a diagnostic test for autism.
But what’s happening on a functional level? A study published this week zooms out to take a 30,000-foot view, tracking how the brain routes information in children with autism—in much the way airlines and electrical grids are mapped—and assessing the function of the network as a whole.