Archive for blood
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
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin. The healthy fetal hemoglobin compensates for the mutated “adult” hemoglobin that makes red blood cells stiffen and assume the classic “sickle” shape. Normally, fetal hemoglobin production tails off after birth, shut ... Read More about BCL11A-based gene therapy for sickle cell disease passes key preclinical test
A first for CRISPR: Cutting genes in blood stem cells
CRISPR — a gene editing technology that lets researchers make precise mutations, deletions and even replacements in genomic DNA — is all the rage among genomic researchers right now. First discovered as a kind of genomic immune memory in bacteria, labs around the world are trying to leverage the technology for diseases ranging from malaria ... Read More about A first for CRISPR: Cutting genes in blood stem cells
Supercharged marrow transplant: Zebrafish reveal drugs that aid engraftment
Bone marrow transplantation, a.k.a. stem cell transplantation, can offer a cure for certain cancers, blood disorders, immune deficiencies and even metabolic disorders. But it’s a highly toxic procedure, especially when a closely matched marrow donor can’t be found. Using stem cells from umbilical cord blood banked after childbirth could open up many more matching possibilities, ... Read More about Supercharged marrow transplant: Zebrafish reveal drugs that aid engraftment