Research
Can asthma be nipped in the bud?
Worldwide, asthma affects an estimated 300 million people, and is expected to surpass 400 million by 2025, according to the World Health Organization. About 1 in 10 U.S. children have asthma, and research shows that the vast majority of them also have allergy. Could that provide a clue to its prevention? Starting at 2 to ... Read More about Can asthma be nipped in the bud?
Research
A first for CRISPR: Cutting genes in blood stem cells
CRISPR — a gene editing technology that lets researchers make precise mutations, deletions and even replacements in genomic DNA — is all the rage among genomic researchers right now. First discovered as a kind of genomic immune memory in bacteria, labs around the world are trying to leverage the technology for diseases ranging from malaria ... Read More about A first for CRISPR: Cutting genes in blood stem cells
Research
Beyond appearances: Molecular genetics revises brain tumor classification and care
Pomeroy Scott Pomeroy, MD, PhD, is Neurologist-in-Chief at Boston Children’s Hospital. He practices in the Brain Tumor Center and is a member of the F.M. Kirby Neurobiology Center. For almost a century, brain tumors have been diagnosed based on their appearance under a microscope and classified by their resemblance to the brain cells from which ... Read More about Beyond appearances: Molecular genetics revises brain tumor classification and care
Research
Double stem cell transplant and other tools are helping children survive neuroblastoma
When Emily Coughlin complained of a sore knee in May 2009, doctors initially suspected Lyme disease. After antibiotics failed to relieve the pain, Emily was diagnosed with neuroblastoma, a cancer that begins in nerve cells outside the brain, just shy of her fourth birthday. Though neuroblastomia is rare — about 700 new cases occur annually in the ... Read More about Double stem cell transplant and other tools are helping children survive neuroblastoma
Patient Stories
Wine used to toast CGD gene therapy trial linked to decades-long scientific journey
When Brenden Whittaker of Columbus, Ohio, the first patient treated with gene therapy for chronic granulomatous disease (CGD), showed successful engraftment last winter, the gene therapy team lifted glasses for a celebratory toast. The wine they sipped was no ordinary wine. The 2012 Bordeaux blend came from an award-winning California vineyard owned and operated by Robert ... Read More about Wine used to toast CGD gene therapy trial linked to decades-long scientific journey
Clinical Care
Detecting shunt failure in hydrocephalus without imaging or surgery: ShuntCheck
Antonio Venus-Reeve, 14, had his first shunt surgery for hydrocephalus when he was 2½ months old. Born at 25 weeks’ gestation, weighing less than two pounds, he had a serious brain bleed seven days later. As Antonio’s head began to swell with excess fluid, neurologists at Boston Children’s Hospital told his mother, Joanne Venus-Williams, that ... Read More about Detecting shunt failure in hydrocephalus without imaging or surgery: ShuntCheck
Research
DIY pain relief with light-activated local nerve blocks
You’ve just had a root canal or knee surgery — both situations that will likely require some sort of local pain medication. But instead of taking a systemic narcotic with all its side effects, what if you could medicate only the part of your body that hurts, only when needed and only as much as ... Read More about DIY pain relief with light-activated local nerve blocks
Clinical Care
Microbiome therapeutics: 6 takeaways from a MassBio panel
Seeing the surprising success of “poop pills” in gastrointestinal C. difficile infection, pharma companies and startups are embracing the microbiome as a new therapeutic target for an astonishing range of maladies. To learn what pioneering companies in the space are thinking about the hope and the hype, Vector recently attended a panel on microbiome therapeutics ... Read More about Microbiome therapeutics: 6 takeaways from a MassBio panel
Research
Forty years waiting for a cure: ALD gene therapy trial shows early promise
A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread. Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a ... Read More about Forty years waiting for a cure: ALD gene therapy trial shows early promise
Patient Stories
George the unstoppable conquers leg-length discrepancy
George Davies, a senior at Bancroft School in Worcester, Massachusetts, faces every challenge, every obstacle and every situation with a sense of what’s possible. He rows varsity crew, plays the electric guitar, and after two years of independent research he’s off to Scotland’s University of St. Andrews to study biochemistry. Dr. James Kasser, Boston Children’s ... Read More about George the unstoppable conquers leg-length discrepancy