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Researchers Discover Areas in the Brain Where Nicotine Could Disrupt Early Brain Development

Neurons found in the brain stem gives new clue to nicotine, sudden infant death syndrome link. Source: Getty Images.

Researchers at Seattle Children’s Research Institute have discovered that populations of neurons in the brain stem have a previously unrecognized susceptibility to disruption by nicotine during early brain development.

Published in the Journal of Comparative Neurology, their findings offer a clue to how nicotine exposure in utero could have a lasting effect on the brain’s wiring and give rise to negative outcomes like sudden infant death syndrome (SIDS). Read full post »

New Contract Will Advance Tuberculosis Research

A federal contract awarded to Seattle Children’s will establish a new center to study tuberculosis. Source: Getty Images.

Seattle Children’s Research Institute is one of three recipients of $30 million in first-year-funding provided by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to establish centers for immunology research to accelerate progress in tuberculosis (TB) vaccine development.

The awards provide up to seven years of support for three Immune Mechanisms of Protection Against Mycobacterium tuberculosis (IMPAc-TB) Centers to uncover the immune responses needed to protect against TB infection. In addition to Seattle Children’s, other IMPAc-TB centers will be led by the Infectious Disease Research Institute, also in Seattle, and the Harvard T.H. Chan School of Public Health.

On the Pulse learned more about the significant research that will be funded by this award which has the potential to provide up to $83 million to the Cascade IMPAc-TB Center, led by Dr. Kevin Urdahl, a TB researcher in Seattle Children’s Center for Global Infectious Disease Research. Read full post »

Research Reveals How Malaria Parasite Plans Ahead, Preparing Blueprint to Strike in Humans

The parasite that causes malaria waits in the salivary glands of the mosquito until it has the opportunity to move to a human host. New research exploring this stage of the parasite offers findings that could help identify new strategies to protect against malaria. Photo: Getty Images.

Within seconds after an infected mosquito bites, the malaria parasite navigates the host skin and blood vessels to invade the liver, where it will stay embedded until thousands of infected cells burst into the bloodstream, launching malaria’s deadly blood-stage infection.

Now, for the first time, a team from Seattle Children’s Research Institute describes how malaria Plasmodium parasites prepare for this journey as they lie in waiting in the mosquito’s salivary glands. Researchers say this knowledge may help identify new strategies to block transmission of the parasite – a critically important step needed for the eradication of malaria, a disease that continues to sicken over 300 million people and kill an estimated 435,000 people worldwide every year. Read full post »

Genetic Study Evolves Understanding of Common Birth Defects of the Brain

Daniel Sokoloff and his mom, Lara Sokoloff, photographed here when Daniel was younger, were one of 100 families who contributed to a study aimed at understanding common birth defects of the brain.

In the largest genetic study of the most common birth defects of the brain diagnosed during pregnancy, researchers from Seattle Children’s Research Institute say their findings evolve our understanding of brain development. The findings will also change the information given to expecting parents when cerebellar malformations, such as Dandy-Walker malformation and cerebellar hypoplasia, are detected prenatally.

With funding support from the Dandy-Walker Alliance and the Philly Baer Foundation, the results gathered from 100 families provide the most accurate information to date about the genetic and non-genetic causes of birth defects involving the cerebellum. They will also help doctors counsel parents about their child’s prognosis after they’re born and their risk of having another child with the abnormality.

“For those who are pregnant, you certainly don’t want to have a problem with the baby, but if doctors detect a brain abnormality, you want accurate information about what that means,” said Dr. William Dobyns, the senior author on the paper published in the American Journal of Human Genetics and an investigator in the Center for Integrative Brain Research. “It’s the same after birth. If you have a child at risk for developmental challenges, it helps to know the cause and what’s going to happen. This study significantly advances our ability to answer those questions.” Read full post »

Seattle Scientists Teach Tibetan Monks Modern Cell Biology

Summer school is in session for researchers at Seattle Children’s Research Institute, and although there are text books and a final exam, very little else about the biology course taught by Dr. Philip Morgan and his fellow scientist and wife, Dr. Margaret Sedensky, is business as usual. That’s because their students are Tibetan monks and their classroom is at a monastic university in southern India. Read full post »

A Stroke of Genius

Dr. Andy Shih is studying how tiny strokes called microinfarcts develop and impact the developing brain.

Strokes come in many shapes and sizes. In children and adults, strokes often present sudden limb or facial numbness, confusion and dizziness.

But some strokes that cause clots to develop in the small blood vessels of the brain don’t exhibit any symptoms at all. Studies have shown that hundreds to thousands of these small, asymptomatic strokes, known as microinfarcts, likely occur over the course of decades in adult brains and may contribute to cognitive decline as we age. Even less is known about the occurrence and consequences of microinfarcts in young, developing brains.

Enter Dr. Andy Shih, principal investigator at Seattle Children’s Research Institute’s Center for Developmental Biology and Regenerative Medicine. Shih hopes to solve the mystery of microinfarcts by using advanced optical imaging — modeling them in the lab and visualizing their effects in real-time. On the Pulse sat down with Shih to learn more about his work and how he’s applying his discoveries from studying dementia in aging brains to understanding how blood vessels and clots first emerge in the brain. Read full post »

Sonny Stays Strong as Cell Therapy for His Rare Condition Advances

Sonny D’Ambrosio, age 7, here with his parents, was diagnosed with an autoimmune condition called IPEX as an infant.

Earlier this year, Nicole D’Ambrosio found herself in front of a room full of scientists that were gathered in part to discuss their progress on a novel cell therapy that has the potential to one day save her son’s life.

She had been asked to present her family’s story as part of a company-wide meeting at Casebia Therapeutics in Boston. As she began, she recounted how her only child, 7-year-old Sonny, has reached the brink of death more times than she can remember because of the rare autoimmune disorder he was diagnosed with as an infant. How the bone marrow transplant he received was the only thing that could save him, but caused endless complications, including skin necrosis and epilepsy.

How the thought of going through another transplant when the initial transplant failed compelled her and her husband to pack up their home and move 3,000 miles west to seek other options. How she lies awake at night praying that Sonny’s body can stay strong enough until a safer treatment comes along. How, despite everything he’s been through, Sonny is still a happy little boy with a wicked sense of humor. Read full post »

‘Like Looking at a Miracle’: Baby Blossoms Thanks to Gene Therapy

Arabella Smygov, 7 months, of Lynnwood, Washington was one of the first babies in the state to receive the gene therapy, Zolgensma. The U.S. Food and Drug Administration approved Zolgensma for the treatment of Spinal Muscular Atrophy in children less than 2 years of age this month.

When Arabella Smygov was diagnosed with spinal muscular atrophy (SMA) type 1 at 3 months old, the first recommendation Dr. Fawn Leigh, a neurologist at Seattle Children’s, gave her parents, Sarah and Vitaliy, was to wait on searching for information about SMA online.

This is because up until a few years ago, SMA type 1 was a fatal diagnosis. Most of the information available online painted a bleak picture. Babies diagnosed with SMA type 1, the most severe and common form of the neurodegenerative disease, usually don’t survive beyond age 2 and if they do, they require full support for breathing from a ventilator.

Leigh had good reason for wanting her parents to have hope for Arabella’s future. Two treatments, including the first-ever drug approved for the condition by the U.S. Food and Drug Administration (FDA) in 2016 called Spinraza, and Zolgensma, a gene therapy approved by the drug agency this month, are rapidly changing the trajectory for children with Arabella’s condition.

“I always remember back to when I had to offer my first SMA diagnosis,” Leigh said. “I was heartbroken to tell this young couple that we didn’t have anything for their baby. Now, we’re planning a future for these babies because we have not one, but two good treatment options.” Read full post »

Makenzie Dances Again After Pediatric Stroke

Makenzie Childs, 6, has returned to doing what she loves most – competitive dance – following a stroke in October 2017.

The lopsided smile on Makenzie’s face said it all. She had just thrown up, and her dad, Shawn Childs, was helping her get cleaned up in the bathtub.

“My husband yelled for me to come into the bathroom and then asked Makenzie to smile,” Jamie Childs, Makenzie’s mom, said. “The smile was devastating. It was only the right half of her face. I knew something wasn’t right.” Read full post »

Researchers Ready B Cells for Novel Cell Therapy

Dr. Richard James is leading research to edit human B plasma cells to act as cell factories capable of delivering sustained, high doses of a therapeutic protein.

Scientists at Seattle Children’s Research Institute are paving the way to use gene-edited B cells – a type of white blood cell in the immune system – to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children’s in as soon as five years. Read full post »