Archive for hematology
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
Too many blood cells: Probing a blood cancer’s genetic origins
At age 7, Meredith Shah began having debilitating migraine headaches. “I would have trouble seeing and shapes were blurry,” she recalls. “It was really painful.” Over time, the frequency and intensity of the headaches escalated. Her parents, Heidi and Nil, sought the help of multiple specialists. But they received few answers, other than an indication ... Read More about Too many blood cells: Probing a blood cancer’s genetic origins
Gene therapy’s future may be all about the bases
Gene therapy offers the possibility of a cure for many genetic disorders, especially those involving a single gene. The first kind of gene therapy used a virus to carry a corrected copy of the gene into people’s cells. When the early viral vectors used in the 1990s were found to have off-target effects, sometimes even ... Read More about Gene therapy’s future may be all about the bases
Tracking an organism’s development, cell by cell
A new mouse model allows scientists to track every cell in the body — from the embryo stage until adulthood. The system is the first of its kind and should yield a greater understanding of development, aging, and disease. Scientists described it last week in the journal Cell. “The dream of many developmental biologists for ... Read More about Tracking an organism’s development, cell by cell
Adults with cancer appear more vulnerable to COVID-19
Cancer, particularly metastatic cancer, can be added to the list of medical conditions that pose a significant risk of worse COVID-19 outcomes , suggests a new study. Led by Boston Children’s Hospital and Zhongnan Hospital of Wuhan University, it represents the first large cohort study to examine COVID-19 outcomes in cancer patients. Findings were published ... Read More about Adults with cancer appear more vulnerable to COVID-19
Following blood stem cells in the body could yield better, safer treatments
Blood stem cells make all the different kinds of blood and immune cells in our body. Scientists have long been trying to make these cells in the lab and use them to make different blood cell types on demand. Mastering these feats could help make bone marrow transplants more efficient and less toxic, and could ... Read More about Following blood stem cells in the body could yield better, safer treatments
Going into science: Women scientists at Boston Children’s offer advice to girls
In honor of the International Day of Women and Girls in Science (February 11), we invited women scientists at all stages of their careers at Boston Children’s Hospital to share their scientific agendas. Here is some of what they had to say. The scientists also offered their advice for girls interested in entering the field. ... Read More about Going into science: Women scientists at Boston Children’s offer advice to girls
After 80 years, genetic causes of Diamond-Blackfan anemia come into view
In 1938, Louis K. Diamond, MD, and Kenneth Blackfan, MD, at Boston Children’s Hospital described a severe congenital anemia that they termed “hypoplastic” (literally, “underdeveloped”) because of the bone marrow’s inability to produce mature, functioning red blood cells. Eighty years later, the multiple genetic origins of this highly rare disease, now known as Diamond-Blackfan anemia, or ... Read More about After 80 years, genetic causes of Diamond-Blackfan anemia come into view
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