Archive for blood disorder
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?
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?
I’ve been there, too: What my baby’s tumor taught me as a NICU nurse
I had a toddler at home when I found out I was pregnant with my twins, Hannah and Sophie. Since I had already had a baby, I thought I knew what to expect during my pregnancy. I also work in the neonatal intensive care unit (NICU) at Boston Children’s Hospital, so I thought I was ... Read More about I’ve been there, too: What my baby’s tumor taught me as a NICU nurse
‘Mom, my brain feels better.’ One mother’s story of her daughter’s fight with epilepsy
Liliane has a lot to be grateful for this holiday season. Until just this year, her 16-year-old daughter Emily, who has epilepsy, suffered relentless seizures that left her temporarily unable to speak or stand. The seizures began when Emily was 4, and living her life around them was all she knew. But today, Emily is ... Read More about ‘Mom, my brain feels better.’ One mother’s story of her daughter’s fight with epilepsy
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
Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored by bluebird bio. Investigations later concluded that the gene therapy delivery vectors were likely not ... Read More about Sickle cell disease, gene therapy, and blood cancers: Mysteries remain
Surviving stem cell transplant: New hope when the donor isn’t a full match
To see Tara Daniels today, with a corporate job in marketing and about to close on a house, you’d never know what she’s been through, how thrilled she is to be alive. This month marks five years since she received a high-risk bone marrow transplant for a life-threatening blood cancer. Tara woke up feeling sick ... Read More about Surviving stem cell transplant: New hope when the donor isn’t a full match