It was a big year for children’s health: We celebrated the U.S. Food and Drug Administration’s approval of the world’s first chimeric antigen receptor (CAR)-T cell therapy this September, followed closely by approval of the very first gene therapy to treat inherited blindness this month — both of which have their roots at Children’s Hospital of Philadelphia and the University of Pennsylvania. But besides the big headline-making breakthroughs (brilliant as they are), we wanted to know what other stories captivated our readers in 2017.
Category Archive: Center for Mitochondrial and Epigenomic Medicine
Patients and families dealing with the rare, debilitating disease Friedreich’s Ataxia showed their true strength at the 10th Annual Friedreich’s Ataxia Symposium held in King of Prussia, Pa. The FA community is a steadfast supporter of research, and they have been gathering for a decade to spend a day with each other building friendships and learning the most up-to-date information on the therapeutic approaches and scientific studies being conducted in the field of FA.
The human genome provides a precise, biological blueprint of life. To implement this blueprint correctly, the genome must be read with great precision, but it’s impossible for this process to be completely error-free. Mistakes during transcription — random errors in how DNA sequences are copied for a gene to be expressed — can happen any time in any number of ways.
September marks National Childhood Cancer Awareness Month, and this year at Children’s Hospital of Philadelphia, we kick-started the commemorative period on the heels of exciting news about breakthroughs in pediatric cancer immunotherapy research. Oncology investigators at CHOP also got a big boost in research funding from Hyundai’s nonprofit organization, Hope on Wheels. And that’s only the beginning: Since September marks the return of the football season, we’re thrilled to share the latest headlines on how the National Football League (NFL) is helping to drive concussion research.
Differences in mitochondrial function are a major factor in understanding the origins of autism spectrum disorders (ASD), according to a new study led by Douglas Wallace, PhD, director of the Center for Mitochondrial and Epigenomic Medicine at Children’s Hospital of Philadelphia, that points way back to genetic vulnerabilities accumulated during ancient human migrations.
Like the mitochondria that Douglas Wallace, PhD, has dedicated his scientific career to studying, energy keeps building about his exciting work. Dr. Wallace’s latest high-profile achievement is being selected as the recipient of the Dr. Paul Janssen Award for Biomedical Research.
A march, a medal, and a media blitz: In this week’s research news, Children's Hospital of Philadelphia investigators made their mark in the history of progressive science in big, bold ways.
Buckle your seatbelts because this has been a busy week for research news at Children’s Hospital of Philadelphia.
Yippee! Hooray! Woo-hoo! We’d like to take a few moments to celebrate the recent accolades given to scientists and physician researchers at The Children’s Hospital of Philadelphia Research Institute who are featured in the latest issue of Bench to Bedside, our monthly newsmagazine.
Welcome back to another weekly roundup of research news from The Children’s Hospital of Philadelphia! As we head into Memorial Day weekend in the U.S. and the informal start to summer, let’s begin with news this week that resonates with some of the things we love about summer.