Archive for blood
Following clinical trial, boy with Fanconi anemia transfusion free
Seven-year-old Ervis of Chicago, Illinois, is a model student with a positive attitude and a megawatt smile. His mom Ofelia calls him “un encantador” — “a charmer,” but life as Ervis knows it is not exactly charmed. Born with Fanconi anemia (FA), a rare hereditary blood disorder that can lead to bone marrow failure and cancer, ... Read More about Following clinical trial, boy with Fanconi anemia transfusion free
Breaking gender biases: What is it like to be a girl with severe hemophilia?
In human biology class this past semester, Morgan DiPrima viewed a PowerPoint presentation that made her head spin. It included a hemophilia slide solely focused on men. “So, I asked the professor if I could do a class presentation on hemophilia for extra credit.” Very few college students “request” extra work, but this 19-year-old Dean ... Read More about Breaking gender biases: What is it like to be a girl with severe hemophilia?
New stem cell pipeline makes more lifelike cell products
Researchers in many fields of science and medicine engineer different types of human cells for drug testing and other purposes. They often begin with stem cells — unspecialized cells that have the potential to form many different types of tissue. By turning on certain genes, the scientists attempt to coax the stem cells into becoming ... Read More about New stem cell pipeline makes more lifelike cell products
Genetics drive deep investigations into blood cell production
Knowing how different kinds of blood cells form from their stem cell progenitors can shed light on blood disorders and aid in finding new treatments. A series of recent studies co-led by researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center applied a variety of genetic tools to provide new insights on blood cell production. ... Read More about Genetics drive deep investigations into blood cell production
Blood stem cell transplants from any donor, without toxicity?
Many blood disorders, immune disorders and metabolic disorders can be cured with a transplant of hematopoietic (blood-forming) stem cells, also known as bone marrow transplant. But patients must first receive high-dose, whole-body chemotherapy and/or radiation to deplete their own defective stem cells, providing space for the donor cells to engraft. These “conditioning” regimens are highly ... Read More about Blood stem cell transplants from any donor, without toxicity?
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San Diego. Manny ... Read More about Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
“Teenage” red blood cells could hold the key to a malaria vaccine
Malaria parasite infection, which affects our red blood cells, can be fatal. Currently, there are about 200 million malaria infections in the world each year and more than 400,000 people, mostly children, die of malaria each year. Now, studying blood samples from patients treated for malaria at a clinical field station in Brazil’s Amazon jungle, ... Read More about “Teenage” red blood cells could hold the key to a malaria vaccine
Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Boston Children’s Hospital is now enrolling patients age 3 to 35 in a clinical trial of gene therapy for sickle cell disease. Based on technology developed in its own labs, it differs from other gene therapy approaches by having a two-pronged action. It represses production of the mutated beta hemoglobin that causes red blood cells to ... Read More about Sickle cell gene therapy to boost fetal hemoglobin: A 70-year timeline of discovery
Medical milestone: Making blood stem cells in the lab
Pluripotent stem cells can make virtually every cell type in the body. But until now, one type has remained elusive: blood stem cells, the source of our entire complement of blood cells. Since human embryonic stem cells (ES cells) were isolated in 1998, scientists have tried to get them to make blood stem cells. In ... Read More about Medical milestone: Making blood stem cells in the lab
Stem cell workaround cracks open new leads in Diamond Blackfan anemia
Diamond Blackfan anemia (DBA) has long been a disease waiting for a cure. First described in 1938 by Louis K. Diamond, MD, of Boston Children’s Hospital and his mentor, Kenneth Blackfan, MD, the rare, severe blood disorder prevents the bone marrow from making enough red blood cells. It’s been linked to mutations affecting a variety of ... Read More about Stem cell workaround cracks open new leads in Diamond Blackfan anemia