Archive for blood
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
Tagged: anemia, blood, blood disorder, gene therapy, hematology
Blood donations help Kit manage Diamond-Blackfan anemia — so she can dance, sing, and enjoy life
Every month, Kit Murdoch needs a blood transfusion to stay alive. The 2-year-old has Diamond-Blackfan anemia, a rare genetic blood disorder that, if untreated, prevents blood from delivering oxygen through the body. While it has been a devastating diagnosis for Kit’s parents, they’re grateful for the specialized care she receives and are constantly amazed that people ... Read More about Blood donations help Kit manage Diamond-Blackfan anemia — so she can dance, sing, and enjoy life
Tagged: anemia, blood, blood disorder, heart, rare disease, ventricular septal defect
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
Tagged: blood, blood disorder, gene editing, gene therapy, hematology, sickle cell disease
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
Tagged: blood, blood disorder, diet, hematology, research rising stars, vascular biology
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?
Tagged: blood, blood disorder, hematology, stem cell transplant, stem cells
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
Tagged: biomarkers, blood, coronavirus, diagnostics, proteomics
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?
New work transforms our knowledge of how blood is formed
The origins of our blood may not be quite what we thought. In groundbreaking research, scientists in the Stem Cell Program at Boston Children’s Hospital used cellular “barcoding” techniques in mice to track the development of blood in real time — and found that blood cells originate not from one type of mother cell, but ... Read More about New work transforms our knowledge of how blood is formed
Tagged: blood, blood disorder, cancer, hematology, stem cell transplant, stem cells
A clinical trial aims to reduce complications in life-saving lung and heart treatment
Extracorporeal membrane oxygenation (ECMO) can be lifesaving for critically ill children with severe lung and heart failure who are not responsive to conventional treatments. This approach takes over the work of those vital organs, enabling patients to rest and recover. It transfers blood from the body into a pump-and-oxygenator machine, where blood is oxygenated and carbon ... Read More about A clinical trial aims to reduce complications in life-saving lung and heart treatment
Tagged: blood, heart, heart center
New findings show risk of bleeding and clotting after COVID-19
A year after COVID-19 was first identified, we have some answers about how to prevent and treat this illness, but there are still many questions. Some of the unknowns include what longer-term effects COVID-19 infection may have on the heart and other systems in the body. One of the complications associated with COVID-19 infection is ... Read More about New findings show risk of bleeding and clotting after COVID-19
Tagged: blood, cardiac research, cardiac surgery, coronavirus, heart, heart center, research