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Milestones & Multidisciplinary Science Make Our Most Engaging Stories of 2024

We tallied up the most popular Cornerstone blog stories of 2024 based on data across our CHOP Research social media platforms.
Amidst a year of change, one thing remains the same: the enthusiasm showed by our Children's Hospital of Philadelphia research community. As in previous years, we tallied up the most popular Cornerstone blog stories of 2024 based on data across our CHOP Research social media platforms.
Our followers on Facebook, LinkedIn, and Twitter/X liked, commented, and shared stories about major milestones like the 20-year anniversary of our Center for Injury Research and Prevention and the 30-year path to a hemophilia gene therapy. Multidisciplinary collaborations also made the top spots, as readers loved our coverage of an investigational gene therapy for hereditary hearing loss and the marriage of big data with emergency department triage.
Science Meets Sashes for Miss Pennsylvania USA 2024, a Woman in STEM

CHOP research tech Noni Diarra represented Pennsylvania at Miss USA 2024.
This past August, Noni Diarra, research technician in CHOP's Center for Data Driven Discovery in Biomedicine (D³b), represented Pennsylvania in the Miss USA 2024 pageant. Diarra's story, part of our "Off Campus" blog series, was about more than just poses and pageant walks. The 23-year-old North Philly native's goal is to advocate for diversity and empower young Black women to achieve their dreams — whether that's pursuing a STEM career or being a voice for their community. Her story resonated with our followers on CHOP Research social media platforms.
"It's not just about the outside optics," Diarra said of her role in Miss USA 2024. "It's also about your entire character and your purpose for being there. It's how you interact with the judges, how you express yourself and your beliefs and values, how you articulate your 'why' – that is the most important part."
Diarra currently works in the Pre-clinical Laboratory Research Unit of D³b, one of CHOP's most cutting-edge collaborative research centers. As a child admitted to CHOP for frequent asthma exacerbations, she was inspired to pursue science and medicine after participating in CHOP's Community Asthma Prevention Program. Moving forward, Diarra hopes to earn her PhD and return to CHOP to continue working in the cancer biology space.
Learn more on Cornerstone about Diarra's journey and experience at CHOP.
Looking Back and Ahead: 25 Years of Preventing and Researching Childhood Injuries

CIRP celebrated 25 years of preventing and researching childhood injuries.
It's hard to believe the Center for Injury Research and Prevention (CIRP), CHOP's very first Center of Emphasis, is celebrating 25 years and beyond! The unique multidisciplinary center has made incredible advances in injury research from driver safety to violence prevention, concussions to rehabilitation biomechanics, and so much more.
We celebrated CIRP's milestones with a Cornerstone story highlighting the Center's future directions, including a new leadership team: Scientific Director Kristy Arbogast, PhD; Associate Director Rachel Myers, PhD; Strategy Director Suzanne Hill; and Business and Administrative Director Ronni Kessler.
The story also touched on how CIRP's strategic priorities have evolved to meet the needs of children and to reflect a diversified patient population.
"What stands out to me about the work at CIRP — and what we want to continue to do as we look forward — is the respect and incorporation of diversity in our research, so that we can make sure we're asking the right questions and partnering with the right people," Dr. Myers said. "This is both within our teams and with our external partners, to bridge academic, clinical, and community expertise in understanding urgent problems and developing feasible solutions in pediatric injury prevention."
Read more on Cornerstone about the impact and vision of CIRP.
CHOP Surgeon Performs First Gene Therapy in Clinical Trial for Hereditary Hearing Loss

An 11-year-old patient received the first gene therapy procedure for hereditary hearing loss performed at CHOP.
In 2023, an 11-year-old clinical trial participant at CHOP received the first gene therapy procedure for hereditary hearing loss. On top of its implications for individuals with hearing loss caused by genetic mutations, the successful surgery heralds yet another scientific advance for gene and cell therapy.
CHOP was one of four clinical trial sites in the world participating in this Phase I/Phase II clinical research trial of AAVAnc80-hOTOF gene therapy, in collaboration with the precision medicine company Akouos.
"Gene therapy for hearing loss is something that physicians and scientists have been working toward for over 20 years," said John Germiller, MD, PhD, an attending surgeon and Director of Clinical Research in the Division of Otolaryngology at CHOP. "While the gene therapy we performed in our patient was to correct an abnormality in one very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss."
Discover more about this potential precision genetic medicine on Cornerstone.
CHOP Researchers Awarded Cancer Grand Challenges Funding

CHOP researchers are part of two global teams awarded Cancer Grand Challenges funding.
In 2024, CHOP scientists were part of two global teams awarded funding from Cancer Grand Challenges, an initiative created by the National Cancer Institute and Cancer Research UK. Team KOODAC and Team MATCHMAKERS are part of five groups selected from 176 initial applicants to receive grants of up to $25 million. On Cornerstone, we highlighted the CHOP researchers on these teams and the innovative projects underway to find treatments for some of the most impenetrable childhood cancers.
Yael Mossé, MD, CHOP Professor of Pediatrics, leads Team KOODAC, which also includes co-investigators John Maris, MD, the Giulio D'Angio Chair in Neuroblastoma Research; Adam Wolpaw, MD, PhD, an assistant professor of Pediatrics; and researchers at 10 institutions from the United States, Austria, France, Germany, and the United Kingdom, as well as an industry partner, Nurix Therapeutics. Team KOODAC tackles the challenge of solid tumors in children.
Meanwhile, Nikolaos Sgourakis, PhD, associate professor in CHOP's Center for Computational and Genomic Medicine, is part of Team MATCHMAKERS alongside researchers from Germany, the Netherlands, Norway, the U.K., and the U.S. Team MATCHMAKERS investigates how to predict which antigens are recognized by T cells using simple laboratory tests and computational prediction.
Learn more about these unique research projects on Cornerstone.
Nurse Scientist Taps Arcus Data to Assess Pediatric Emergency Triage Accuracy

A CHOP nurse scientist is leveraging the power of data to improve the safety and effectiveness of emergency department triage for children.
Two of CHOP's unique strengths, nursing research and big data, came together in this story about improving emergency department (ED) triage for children. Warren Frankenberger, PhD, RN, received a $100,000 research award from the National Institute of Child Health and Human Development to determine the accuracy of the Emergency Severity Index (ESI), which assesses and prioritizes the level of urgent care a child receives in the ED.
The project builds on Dr. Frankenberger's previous collaboration between the CHOP Center for Pediatric Nursing Research and the CHOP Research Institute Arcus team. The Arcus-based research, published in the Journal of Emergency Nursing, assessed ESI's accuracy in more than 300,000 emergency department visits at CHOP using natural language processing technology.
"The findings from Dr. Frankenberger's latest study were integral to this NICHD grant award and demonstrate the importance of interprofessional research collaborations," said Margaret McCabe, PhD, RN, FAAN, senior director, Center for Pediatric Nursing Research and Evidence-Based Practice. "This is just one example of how nurse-led research at CHOP is successfully addressing national challenges in pediatric healthcare that may ultimately lead to improved care delivery and outcomes for children and families."
Read more about Dr. Frankenberger's research project on Cornerstone.
A 30-Year Journey: How CHOP Researchers Designed the Gene Therapy Playbook

The FDA approved a one-time gene therapy for adults with hemophilia B, which was pioneered at CHOP.
In 2024, the U.S. Food and Drug Administration approved a one-time gene therapy for adults with hemophilia B. The treatment, called fidanacogene elaparvovec (Beqvez®, now licensed by Pfizer), can potentially change the lives of patients over 18 who have relied on daily therapeutic injections.
Our Cornerstone coverage highlighted the nearly 30-year path leading up to the approval, from the time Katherine High, MD, one of gene therapy's pioneering proponents, joined CHOP in 1992, to the creation of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, and beyond.
"Dr. High and many at CHOP were fundamental in establishing that first set of rules — the regulatory playbook for gene therapy clinical trials — and those rules, for the most part, remain in place today," said Lindsey George, MD, attending hematologist and director of CHOP's Clinical In Vivo Gene Therapy Group who led the phase 1/2 clinical trial for the treatment. "Years ago, hospital leadership said, 'Gene therapy has tremendous potential to impact pediatric health,' and they stayed true to that vision."