Archive for blood
Tough cookie: Steroid therapy helps Alessandra thrive with Diamond-Blackfan anemia
Two-year-old Alessandra is many things. She’s sweet, happy, curious, and, according to her parents, Ralph and Irma, a budding food critic. “She’s a food snob!” Ralph says, laughing. “She recognizes the expensive pasta, the homemade versus store-bought food.” But above all, Alessandra is resilient, and her parents have known that from the very start: At ... Read More about Tough cookie: Steroid therapy helps Alessandra thrive with Diamond-Blackfan anemia
Unique data revealed just when Mickey’s heart doctors could operate
When Mikolaj “Mickey” Karski’s family traveled from Poland to Boston to get him heart care, they weren’t thinking about pressure-volume (PV) loops. His parents simply wanted him to receive treatment for a complex condition that he couldn’t get back home. Little did they know the mathematical computational power of those PV loops would play a ... Read More about Unique data revealed just when Mickey’s heart doctors could operate
It’s all in the PV loops: New analytical model could improve circulation assessments before heart surgery
The double-switch operation corrects the congenital reversal of the heart’s ventricles and its two main arteries. It’s a practical way of putting the ventricles into the position they belong so that children with congenitally corrected transposition of the great arteries (CC-TGA) can benefit from enhanced circulation. Surgery, though, doesn’t come without risks. Some children’s left ventricles — ... Read More about It’s all in the PV loops: New analytical model could improve circulation assessments before heart surgery
Blood across our lifetimes: An age-specific ‘atlas’ tells a dynamic story
The stem cells that form our blood, also known as hematopoietic stem cells (HSCs), are with us throughout our lives. A new study reveals how HSCs ramp up and pivot their activities depending on the body’s needs at the time, from before we’re born until old age. Researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders ... Read More about Blood across our lifetimes: An age-specific ‘atlas’ tells a dynamic story
A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing
Diamond-Blackfan anemia (DBA), first described at Boston Children’s Hospital in 1938, is a rare blood disorder in which the bone marrow cannot make mature, functioning red blood cells. Children with this life-threatening anemia have few treatment options. A small handful with a well-matched donor can be cured with bone marrow transplant, but most rely on ... Read More about A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing
Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Ed. Note: This post updates an earlier post from 2018. In a landmark decision today, the Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease. One of them, Casgevy, has deep scientific roots at Boston Children’s Hospital — and is also the first therapy using CRISPR gene editing to gain FDA ... Read More about Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
The clot thickens: Kellie Machlus, PhD
Part of an ongoing series profiling researchers at Boston Children’s Hospital. Platelets are the bandages of our blood, forming clots when we sustain an injury. Yet little is known about how they’re made, and there are no drugs that can immediately and directly trigger their production. Boston Children’s Hospital researcher Kellie Machlus, PhD, (@theclotthickens) couldn’t ... Read More about The clot thickens: Kellie Machlus, PhD
Could we make blood anywhere in the body?
Our bodies make blood in a specialized niche — a “nursery” within our bone marrow that nurtures blood stem cells so they can replicate and make different kinds of blood cells. The lab of Leonard Zon, MD, has even shown how blood stem cells, once they settle in the niche, are “cuddled” by nearby cells. ... Read More about Could we make blood anywhere in the body?
Deep plasma proteomics: Back to the future
Blood plasma is collected from people routinely during clinical care and for research. It is potentially a rich source of protein biomarkers for diagnostic and prognostic purposes, for measuring response to treatment, and for revealing disease biology. Yet identifying such biomarkers in plasma with proteomics, our best available tool, has been a challenge: Ninety-nine percent ... Read More about Deep plasma proteomics: Back to the future
Could gene therapy relieve post-hemorrhagic hydrocephalus?
Premature infants, especially very low birthweight babies, are at risk for intraventricular hemorrhage. A frequent complication of these brain bleeds is hydrocephalus, an accumulation of cerebrospinal fluid (CSF) in the brain ventricles that can gravely disrupt brain development. If hydrocephalus develops, a child may need shunt operations throughout life to manage the fluid buildup. Could ... Read More about Could gene therapy relieve post-hemorrhagic hydrocephalus?