Archive for cell therapy
A promising step toward safer, less toxic stem cell transplants
Stem cell transplantation (also called bone marrow transplantation) and gene therapy are among the most powerful treatment approaches for blood diseases such as sickle cell disease, beta thalassemia, immune deficiencies, and some blood cancers. Replacing or correcting the blood-forming stem cells can offer the possibility of long-lasting benefit or a cure. However, before patients can ... Read More about A promising step toward safer, less toxic stem cell transplants
Mitochondrial transfer restores heart muscle — but how?
Transferring mitochondria from a patient’s healthy skeletal muscle to damaged, ischemic heart tissue has been shown to restore heart muscle, increase energy production, and improve ventricular function. After pioneering preclinical work by James McCully, PhD, at Boston Children’s Hospital about a decade ago, cardiac surgeons led by Sitaram Emani, MD, have been testing it as ... Read More about Mitochondrial transfer restores heart muscle — but how?
Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators were a breakthrough for cystic fibrosis, improving the movement of chloride and water and moistening mucus secretions. But these drugs are expensive, don’t work in all patients with cystic fibrosis, and have side effects and interactions with other drugs. People who do respond to CFTR modulators must take ... Read More about Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Research 2022: Tackling disease in new ways
Researchers across Boston Children’s spent 2022 imagining new solutions to old challenges in health and medicine, opening the door to brand-new treatments. Here are a few areas where research is poised to make a difference in children’s — and even adults’ — lives. Rethinking disease through genetics Genetic sequencing is becoming a fundamental tool for discovering ... Read More about Research 2022: Tackling disease in new ways