Boston Children's Research

Congratulations to our own Jordan Farrell for being a CURE Epilepsy award recipient!

Ultrasound-guided fine-needle aspiration (FNA) has long been used as a safe and effective way to evaluate thyroid nodules in adults, usually without sedation. Now, research from Ari Wassner, MD, and others in our Thyroid Center offers powerful evidence that this approach is safe for pediatric patients, too. 👇

How has the standard of care evolved over the last 10 years for status epilepticus, including disparities in timely access to treatment? Learn more from Jennifer Gettings from the Division of Epilepsy and Clinical Neurophysiology and others in this Lancet Neurology literature review. 👇

Athletes often warm up just before they are about to perform, yet similar "just-in-time" training is rare to non-existent in medicine. A recent randomized clinical trial led by Stephen Flynn and others in the Department of Anesthesiology, Critical Care, and Pain Medicine found that coaching inexperienced anesthesiology trainees just before intubating an infant increased their first attempt success rate while adding little burden to the coaching team. The results, published this week in BMJ, provide a proof of concept that could be applied to other high-risk procedures. 👇

mRNA vaccines clearly saved lives during the COVID-19 pandemic, but older people had a somewhat reduced immune response to the vaccines compared with younger adults. Why? Boston Children’s researchers, led by Byron Brook, PhD, and Ofer Levy, MD, PhD, found some answers, while providing proof-of-concept of a new system called MEMPHIS that can model immune responses to vaccines in a dish, potentially expediting efforts to make vaccines even more effective. Learn more about this technology 👇

The new gene therapies for sickle cell disease, such as Casgevy, have been game-changing for the patients that receive them. But Stuart Orkin, whose work led the way to Casgevy, isn’t done yet: He wants a pill that could be made widely available. In work just published in Science, his lab has opened the way to destabilizing the target molecule, BCL11A, pharmaceutically, so that people can make non-sickling fetal hemoglobin. Learn more on our blog 👇

Congratulations Seth Rakoff-Nahoum, M.D., Ph.D., for being among the 2024 class of The Pew Charitable Trusts Innovation Fund investigators. 👏 This award supports collaboration on interdisciplinary research projects that explore foundational questions about human biology and disease. Dr. Rakoff-Nahoum will team up with Jing-Ke Weng, Ph.D., of Northeastern University, to precisely trace food molecules’ pathways, modifications, and host interactions and explore a person’s immune response to food proteins. Their work seeks to expand current knowledge of the relationship between food and the human body beyond nutritional values. 👇

We’re excited that our Ophthalmology department has received a $2M research endowment from Knights Templar Eye Foundation, Inc. Under the eye of Lois Smith, MD, PhD, the funds will help advance clinical and laboratory research on childhood blindness, including the use of enhanced imaging, AI, and data science for research on eye disease; investigating neuroprotective agents to reverse retinopathy; advancing genetic therapies for inherited eye diseases; and exploring cell-based therapies.

Grant Rowe, MD, PhD, and colleagues have created a first of its kind atlas that details properties of human blood formation spanning prenatal development, childhood, maturation to adulthood, and aging. Based on this framework, they created a public web interface to allow the community an easily accessible way to browse the scRNA-seq data from all 58,041 human CD34+ hematopoietic stem and progenitor cells. Learn more in Nature Methods. 👇

Dr. Elizabeth DeWitt and colleagues recently demonstrated the safety and efficacy of intraoperative mapping during complex biventricular congenital heart disease (CHD) operations. Their results, published in The Journal of the American College of Cardiology, make a case for using this procedure to better understand patient-specific anatomy and thus reduce postoperative complications for patients with CHD and heterotaxy syndrome.