Weight loss: When scientific paradigms collide
David S. Ludwig, MD, PhD, co-directs the New Balance Foundation Obesity Prevention Center at Boston Children’s Hospital. Because conventional thinking usually rests on years (sometimes centuries) of research, scientists are naturally skeptical of radical new ideas. But occasionally, problems with normal science arise, such as a persistent failure to solve important puzzles and anomalies that can’t be ... Read More about Weight loss: When scientific paradigms collide
Making ready-made CAR T cells for cancer immunotherapy
In CAR T-cell immunotherapy, T cells from a patient’s own blood are engineered to carry so-called chimeric antigen receptors (CAR) that enable the T cells to attack and kill tumor cells. While CAR-T therapy is a powerful approach for certain leukemias and lymphomas, it’s not available for many patients who need it. It can be ... Read More about Making ready-made CAR T cells for cancer immunotherapy
A new vaccine formulation could finally protect babies against RSV
Though often mild, respiratory syncytial virus (RSV) infection can cause babies to be hospitalized with bronchiolitis or pneumonia. Globally, it is the leading cause of death in children under 5. Several vaccines against RSV are being tested in adults. But there has been no progress on an RSV vaccine for children since 1966 — the ... Read More about A new vaccine formulation could finally protect babies against RSV
A potential danger of CRISPR gene editing — and why base editing may be safer
Gene therapy using CRISPR/Cas9 gene editing is currently in clinical trials around the world for a variety of diseases, including various cancers, blood disorders, and metabolic disorders. It works by making cuts in DNA — both strands of the double helix — to insert or remove genes. But CRISPR carries a potential, previously undiscovered danger, ... Read More about A potential danger of CRISPR gene editing — and why base editing may be safer
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
Exploiting a vulnerability in an aggressive leukemia
Survival has improved greatly in children with acute lymphocytic leukemia (ALL). But a certain form of ALL that occurs mostly in babies is still very lethal, with a survival rate below 50 percent: B-cell acute lymphoblastic leukemia with rearrangements of the mixed lineage leukemia gene, or MLL B-ALL. “Something about the biology of this type ... Read More about Exploiting a vulnerability in an aggressive leukemia
Getting to the heart of heart muscle function
Every heart muscle cell, or cardiomyocyte, is studded with tiny, intricate structures called dyads. The dyads are like orchestra conductors: They coordinate incoming electrical signals with release of calcium in the muscle, triggering contraction. When dyads work properly, the different segments of heart muscle contract in unison; when they don’t, heartbeats may be too weak ... Read More about Getting to the heart of heart muscle function
Cell therapy for lung disease? Proof-of-concept study shows promise
Many serious pulmonary diseases, including genetic lung diseases, lack an effective treatment other than the most extreme: lung transplant. A team at Boston Children’s Hospital envisions a much better option: cell therapy, using lung stem cells created from patients’ own cells to repair or replace damaged lung tissue. For patients with genetic lung diseases, the ... Read More about Cell therapy for lung disease? Proof-of-concept study shows promise
A new angle on the cause of Alzheimer’s disease: Accumulating brain mutations
Alzheimer’s disease is marked by a loss of functional neurons in the brain. But what causes this loss? A new study reveals that people with Alzheimer’s have an abundance of newly acquired mutations in their neurons — more than people of the same age without Alzheimer’s, and enough to disable genes important to brain function. ... Read More about A new angle on the cause of Alzheimer’s disease: Accumulating brain mutations
Beyond fluid buildup: Rethinking congenital hydrocephalus
Hydrocephalus is classically seen as a plumbing problem, the result of too much cerebrospinal fluid (CSF) in the brain or dysregulation of fluid flow. It is usually treated with shunts to drain the CSF, or with ETV/CPC, which makes a small hole to drain the CSF and burns off the choroid plexus tissue that produces ... Read More about Beyond fluid buildup: Rethinking congenital hydrocephalus