Tag Archive: Perelman School of Medicine

Nov 9 2018

How Can Gene Editing Breakthroughs Help Prevent Congenital Disease Before Birth?

The findings:

In a first-of-its kind study, scientists performed prenatal gene editing in animals to prevent a lethal metabolic disorder and effectively open the door for similarly treating congenital diseases in humans before birth. Using gene editing technology, the team successfully targeted a gene that regulates cholesterol levels to lower cholesterol and, additionally, turned off the effects of a mutation that causes a lethal liver disease called hereditary tyrosinemia type 1 (HT1) in mice.

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Nov 2 2018

In the News: Fertility After Childhood Cancer, Restoring Vision, NYAS Podcast, Obesity and Autism, Genes in Childhood Epilepsy

The scientific wonder of stem cell research and its implications for medicine have come a long way in the last decade: At Children’s Hospital of Philadelphia Research Institute, our investigators’ innovative use of stem cell science to approach complex pediatric conditions continues to inspire for their potential to improve outcomes in children’s health. In our latest news roundup, learn about novel stem cell research from our Cancer Center and Division of Urology that aims to preserve the future fertility of boys who undergo childhood cancer treatment. Discover a new project co-led by a CHOP neurology researcher that takes a stem cell approach to restore vision cells in blind dogs.

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Oct 19 2018

In the News: Sepsis in Scientific American, Updating Genetic Tests, Prenatal Gene Editing, Nature Magazine, Type 1.5 Diabetes

Our breakthrough research at Children’s Hospital of Philadelphia Research Institute made headlines in both popular science media outlets and prominent scientific journals alike, from advances in detecting sepsis, to pioneering work in prenatal gene editing, to the future of genomic medicine. Read on to learn more about how our scientists stay at the cutting-edge of their fields by approaching pediatric medicine inventively, whether it’s by developing the most effective alerts in emergency medicine, modernizing genetic testing models to keep up with an accelerating pace of discovery, and beyond. 

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Jul 27 2018

In the News: ROP Screening Tool, Sickle Cell Disease Target, PolicyLab Keynote, 2018 St. Baldrick’s Grants

It’s still a month before teachers and students are officially back to school, but here at Children’s Hospital of Philadelphia Research Institute, you can learn something new every day. In this edition of our biweekly news roundup, discover the latest findings from our ophthalmologists on how clinicians should choose to screen premature babies for a potentially blinding eye disorder, find out how CRISPR-based technology allowed scientists to reveal insights into sickle cell disease, and prepare for an educational and exciting speech from the recently announced keynote speaker at PolicyLab’s upcoming 10th Anniversary Forum

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Apr 13 2018

Entrepreneurial Science Scholars Turn Ideas Into Medical Innovation

By definition, entrepreneurs are energetic leaders who challenge existing ideas to drive impactful change. Entrepreneurs think outside the box, follow their passion, and stay resilient and resourceful to achieve their goals. At Children’s Hospital of Philadelphia, fellows in the Entrepreneurial Science Scholars Program do all of these things — and more — to improve the health of children and families.

On Feb. 22, we celebrated this year’s CHOP Entrepreneurial Science Scholars, a group of six clinician-researchers who are conducting pioneering research and innovation in diverse and critical fields. The CHOP Entrepreneurial Science Scholars Program aims to produce highly trained investigators skilled in translational research and the generation of creative solutions to biomedical problems. Joseph St. Geme, MD, Physician-in-Chief and Chairman of the Department of Pediatrics at CHOP, hosted the event.

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Apr 6 2018

U.S. News & World Report, David Barrett, Driving & ADHD, Bike Safety, Concussion Research

Outdoor sports, biking, and bustling streets might be welcome signs of warmer weather and longer days, but they’re also research topics studied rigorously by investigators at our Center for Injury Research and Prevention (CIRP) in their quest to ensure safer environments for children and families. In this week’s roundup of Children’s Hospital of Philadelphia research headlines, learn about a new study from CIRP into the various causes and mechanisms of youth concussions beyond contact sports, discover cool technology that allows scientists to study how cyclists move and make decisions on urban streets, and find out how the CIRP driving simulator is helping to advance what we know of teen driving behaviors. On top of that, we congratulate the Cancer Center’s Dr. David Barrett on a new award from Stand Up to Cancer and offer big congratulations to our Department of Pediatrics’ continued success!

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Apr 3 2018

CHOP Ranked Nation's Top Pediatrics Department for 2019

With enormous pride and admiration, we are thrilled to share the news that for the sixth year in a row, U.S. News & World Report has ranked the Department of Pediatrics at Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania first place in pediatric medical education — a top spot shared with Harvard University this year. Once again, the high honor illustrates the excellent commitments our faculty make every day in training physicians and researchers for a future filled with breakthroughs. Congratulations to our amazing Pediatrics Department!

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Mar 21 2018

Lifespan Brain Institute Symposium Digs Into Roots of Mental Illness

Leaders of the Lifespan Brain Institute (LiBI) brought together experts in child and adult psychiatry, and basic and translational science, to delve into the origins of mental illness, during the Institute’s first symposium, “Pathological Antecedents to Neuropsychiatric Disorders.” Throughout the day, 200 attendees learned about how the typical trajectory of brain development and function is derailed in psychiatric disorders at various points throughout life — perhaps as early as in the womb.

LiBI is uniquely positioned as a broad collaboration between Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania that supports research across the fetal-adult continuum, which is a pillar of CHOP Research Institute’s strategic plan.

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Jan 11 2018

Is Autism Associated With Stronger or Weaker Brain Connections?

The Finding:

In a new study that sheds light on a longstanding paradox in autism research, investigators showed how children and teens with autism spectrum disorder (ASD) can have both stronger and weaker brain connections than their typically developing peers. Resting-state functional magnetic resonance imaging (fMRI) revealed that while individuals with ASD have weaker brain connections compared to their peers overall (a measure known as absolute connectivity), they simultaneously exhibited stronger connections within the brain networks implicated in attention and social cognition compared to their other networks (a measure known as relative connectivity). Additionally, the more these brain connections differed from typical development, the more severe the child’s ASD symptoms seemed to be.

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Dec 27 2017

Our Most Read Stories of 2017 Bring Back the Wonder of Childhood

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.

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