Archive for blood disorder
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
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 small act of kindness: Blood donations get Sadie off the sidelines after her aplastic anemia diagnosis
In March of 2024, Sadie’s life was interrupted. A busy high school senior with classes to attend, soccer matches to play, and a much-anticipated trip to France on the horizon, Sadie was very active and felt generally healthy. But when a follow-up appointment at her pediatrician’s office led to an urgent referral to Boston Children’s ... Read More about A small act of kindness: Blood donations get Sadie off the sidelines after her aplastic anemia diagnosis
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
Curbing blood cancers by teaching immune cells to kill mutant stem cells
Blood stem cells, which give rise to all of our blood cell types, undergo a quality assurance process after they’re born. As the lab of Leonard Zon, MD, director of the Stem Cell Research program at Boston Children’s, has documented, immune cells known as macrophages interact with each newly born cell. They engulf and eat ... Read More about Curbing blood cancers by teaching immune cells to kill mutant stem cells
In a thriving gene therapy program, nursing leadership is the driving force
Gene therapy was made possible by decades of technological advances. But to execute gene therapy at scale? That would not be possible without the foresight, organization, and innovation of nursing leadership at Boston Children’s Hospital. The gene therapy implementation framework they have built has become a gold standard for the field. Founded in 2010, the ... Read More about In a thriving gene therapy program, nursing leadership is the driving force
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
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 the right dietary fat help boost platelet counts?
Aside from transfusions, there currently is no way to boost people’s platelet counts, leaving them at risk for uncontrolled bleeding. Could something as simple as a dietary change raise platelet counts in people with low levels, such as cancer patients receiving chemotherapy? New science out of the lab suggests that the answer might be yes. ... Read More about Could the right dietary fat help boost platelet counts?