10 posts · 12,807 views
People who rely on protein-based drugs often have to endure IV hookups or frequent injections, sometimes several times a week. And protein drugs – like Factor VIII and Factor IX for patients with hemophilia, alpha interferon for hepatitis C, interferon beta for multiple sclerosis — are very expensive.
What if they could be made by people’s own bodies?
Combining tissue engineering with gene therapy, researchers at Children’s Hospital Boston showed that it’s pos........ Read more »
Fliesler, Nancy. (2011) Avoiding the needle: Engineering blood vessels to secrete drugs. Vector, A Children's Hospital Boston Blog. info:/
Low-grade inflammation caused by obesity is widely believed to contribute to insulin resistance and type 2 diabetes. But, as it turns out, inflammation activates two proteins that appear critical for maintaining good blood sugar levels. Reporting in Nature Medicine, endocrinology researcher Umut Ozcan demonstrates that activating either of these proteins artificially can normalize blood sugar in severely obese and diabetic mice.
That’s a completely new way of looking at diabetes, and su........ Read more »
Lee J, Sun C, Zhou Y, Lee J, Gokalp D, Herrema H, Park SW, Davis RJ, & Ozcan U. (2011) p38 MAPK-mediated regulation of Xbp1s is crucial for glucose homeostasis. Nature Medicine. PMID: 21892182
We all know the problem: The cost of health care needs to come down. About five years ago, pediatric cardiologists at Children’s Hospital Boston realized it was critical to practice more cost-effectively. “Most of the money that is going to be removed from the federal budget to reduce budgetary deficits is going to come from health care in one fashion or another,” cardiologist-in-chief James Lock told an audience of senior Children’s physicians last month. “There&rs........ Read more »
Shojania KG, Sampson M, Ansari MT, Ji J, Doucette S, & Moher D. (2007) How quickly do systematic reviews go out of date? A survival analysis. Annals of Internal Medicine, 147(4), 224-33. PMID: 17638714
Rathod RH, Farias M, Friedman KG, Graham D, Fulton DR, Newburger JW, Colan S, Jenkins K, & Lock JE. (2010) A novel approach to gathering and acting on relevant clinical information: SCAMPs. Congenital Heart Disease, 5(4), 343-53. PMID: 20653701
Friedman KG, Kane DA, Rathod RH, Renaud A, Farias M, Geggel R, Fulton DR, Lock JE, & Saleeb SF. (2011) Management of pediatric chest pain using a standardized assessment and management plan. Pediatrics, 128(2), 239-45. PMID: 21746719
In the past few decades, what used to be considered medical miracles have become expected and everyday. More children are surviving prematurity, even extreme prematurity. Congenital heart defects are routinely repaired, leukemia has largely become curable, and conditions like sickle cell disease and cystic fibrosis have become manageable chronic conditions with a greatly increased life expectancy.
That’s created a new problem: young adults entering an adult healthcare system that isn&rs........ Read more »
Sawicki GS, Whitworth R, Gunn L, Butterfield R, Lukens-Bull K, & Wood D. (2011) Receipt of Health Care Transition Counseling in the National Survey of Adult Transition and Health. Pediatrics. PMID: 21824879
Just as there’s good cholesterol and bad cholesterol, good carbs and bad carbs, there’s also good fat. Whereas white fat stores energy, padding our hips, thighs, arms and bellies, brown fat — studded with energy generators known as mitochondria – burns energy. Newborns have a ring of brown fat around their necks, helping them stay warm. By adulthood, it’s detectable in only 3 percent of men and 7.5 percent of women, with higher rates among younger and thinner peopl........ Read more »
Drubach LA, Palmer EL 3rd, Connolly LP, Baker A, Zurakowski D, & Cypess AM. (2011) Pediatric Brown Adipose Tissue: Detection, Epidemiology, and Differences from Adults. The Journal of Pediatrics. PMID: 21839465
Every year, the flu tries to outwit humanity. By shifting parts of its outer coat, the virus renders the flu vaccine from the previous year obsolete, bringing another season of misery. And every year, we fight back with a new vaccine, finding a new chink in the virus’s armor and giving ourselves another brief window of protection.
But if Stephen Harrison, chief of Children’s Division of Molecular Medicine, is right, we might be able to train our immune systems to look past the flu........ Read more »
Whittle, J., Zhang, R., Khurana, S., King, L., Manischewitz, J., Golding, H., Dormitzer, P., Haynes, B., Walter, E., Moody, M.... (2011) Broadly neutralizing human antibody that recognizes the receptor-binding pocket of influenza virus hemagglutinin. Proceedings of the National Academy of Sciences. DOI: 10.1073/pnas.1111497108
When your doctor wants to see inside your body, he or she typically chooses from four options: traditional X-ray, computed tomography (CT), ultrasound, or magnetic resonance imaging (MRI). (Yes, there are other methods of clinical imaging, but we’ll stick to the most common for the moment).
Each method has its strengths and weaknesses, largely dependent on what your doctor is looking for. MRI and CT, for instance, give highly detailed pictures of anatomic structures and soft tissues, bu........ Read more »
Penna FJ, Chow JS, Minnillo BJ, Passerotti CC, Barnewolt CE, Treves ST, Fahey FH, Dunning PS, Freilich DA, Retik AB.... (2011) Identifying ureteropelvic junction obstruction by fluorescence imaging: a comparative study of imaging modalities to assess renal function and degree of obstruction in a mouse model. The Journal of Urology, 185(6), 2405-13. PMID: 21511294
In emergency situations involving children, it’s tempting for doctors to do everything possible to get information, especially when anxious parents are at hand. Unfortunately, that can mean a lot of unnecessary imaging and radiation exposure, and sometimes fruitless exploratory surgery.
This has spurred a search for biomarkers that can reliably make or rule out a diagnosis, as in appendicitis, and the creation of decision rules about the need for imaging, as in minor head trauma and blu........ Read more »
Neuman MI, Graham D, & Bachur R. (2011) Variation in the use of chest radiography for pneumonia in pediatric emergency departments. Pediatric Emergency Care, 27(7), 606-10. PMID: 21712748
Neuman MI, Scully KJ, Kim D, Shah S, & Bachur RG. (2010) Physician assessment of the likelihood of pneumonia in a pediatric emergency department. Pediatric Emergency Care, 26(11), 817-22. PMID: 20944506
Neuman MI, Monuteaux MC, Scully KJ, & Bachur RG. (2011) Prediction of Pneumonia in a Pediatric Emergency Department. Pediatrics. PMID: 21746723
In the 40 years of the war on cancer, there is probably no greater success story than that of childhood leukemias. Once nearly uniformly fatal, some forms of acute lymphoblastic (ALL) and acute myeloid (AML) leukemias can now be cured in 80 or even 90 percent of cases.
The prognosis for the remaining 10 to 20 percent is not as good, especially if the cancer involves a reshuffling or rearrangement of the mixed lineage leukemia (MLL) gene. “We still only achieve about 50 percent success i........ Read more »
Bernt KM, Zhu N, Sinha AU, Vempati S, Faber J, Krivtsov AV, Feng Z, Punt N, Daigle A, Bullinger L.... (2011) MLL-Rearranged Leukemia Is Dependent on Aberrant H3K79 Methylation by DOT1L. Cancer Cell, 20(1), 66-78. PMID: 21741597
Daigle SR, Olhava EJ, Therkelsen CA, Majer CR, Sneeringer CJ, Song J, Johnston LD, Scott MP, Smith JJ, Xiao Y.... (2011) Selective Killing of Mixed Lineage Leukemia Cells by a Potent Small-Molecule DOT1L Inhibitor. Cancer Cell, 20(1), 53-65. PMID: 21741596
While the genome’s As, Ts, Cs, and Gs hold the instructions for making proteins, how does a cell know when to read a gene? And could it relate to developmental disorders?
These gene-reading instructions are encoded in our epigenome, a set of factors that give our cells exquisite control over when and where to turn individual genes on and off. This control involves a delicate and complex dance between DNA and proteins called histones – DNA wraps itself around histones to create a c........ Read more »
Iwase S, Xiang B, Ghosh S, Ren T, Lewis PW, Cochrane JC, Allis CD, Picketts DJ, Patel DJ, Li H.... (2011) ATRX ADD domain links an atypical histone methylation recognition mechanism to human mental-retardation syndrome. Nature Structural , 18(7), 769-76. PMID: 21666679
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