The Children's Hospital of Philadelphia Research Institute

The Children's Hospital of Philadelphia Research Institute CHOP Research advances the health of children by turning scientific discovery into medical innovation
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01/21/2026

Watch Dr. Beverly Davidson, Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia, talk about her team's identification of two novel adeno-associated virus (AAV) capsid variants that could improve the cost, dosage, and precision of gene therapy to the brain. "Imagine looking at the night sky in New York City, where you'll see only the moon and a handful of stars," Dr. Davidson said. "That is the current state of gene therapy. What we've created here is like looking at the night sky over the Sahara Desert."

To learn more about our development of unique, leading-edge technologies at CHOP, check out our 2025 Annual Report: http://ms.spr.ly/6189QBeYt

01/12/2026

In this video, Dr. Rebecca Ahrens-Nicklas, Assistant Professor of Pediatrics at Children's Hospital of Philadelphia, talks about one of our biggest breakthroughs of 2025: the treatment of Baby KJ Muldoon with the first-ever personalized gene-editing drug. You can read about Baby KJ's full story, beginning with his diagnosis at CHOP via the Baby Eagle Initiative, in our 2025 Research Annual Report: http://ms.spr.ly/6188tfiWe

šŸ„ 2025 was a big year for breakthroughs that brought years of scientific research to the bedside. In the gallery below, ...
01/02/2026

šŸ„ 2025 was a big year for breakthroughs that brought years of scientific research to the bedside. In the gallery below, read about some of these discoveries from our investigators at Children's Hospital of Philadelphia Research Institute, including the first-ever personalized gene therapy for a rare disease, a novel vaccine to prevent and treat the bacterial infection Clostridioides difficile, and more. You also can read each full story in our 2025 Research Annual Report, now live! http://ms.spr.ly/6181tINlx

Our annual round-up of the most popular stories of #2025 is here! We tallied up the Cornerstone blog posts that receive...
12/31/2025

Our annual round-up of the most popular stories of #2025 is here! We tallied up the Cornerstone blog posts that received the most engagements on our social media platforms to see what research stories resonated the most with you, our community, in 2025. Check out the full list now: http://ms.spr.ly/6184taRNk

12/30/2025

Our 2025 Research Annual Report is live! Click the link in our bio to dive into this celebration of our advances and accomplishments, from cultivating a world-class academic medical research community to developing transformative technologies and treatments, and so much more. From our community to yours, CHOP Research Institute wishes you happy holidays ā­

12/29/2025

2025 was a transformative year at Children's Hospital of Philadelphia Research Institute!

Our scientists shared their thought leadership through global collaborations and personalized approaches to pediatric care.

We expanded our physical footprint with the launch of new institutes and our scientific imprint by establishing innovative research programs and centers of emphasis.

Several groundbreaking therapiesā€”like a first-in-kind gene-editing treatment for a newborn and expanded access to CAR-T therapyā€”demonstrated our continued leadership in advancing rare disease care.

And our researchers developed novel scientific tools and techniques, including a wearable neuroimaging device, deep learning genomic models, and next-generation gene therapy delivery platforms.

We invite you to watch this video with Executive Vice President and Chief Scientific Officer Dr. Susan Furth, or take a moment to read our 2025 Research Annual Report, now available to view online: http://ms.spr.ly/6181taRvv

As the year comes to a close, weā€™re reflecting on the incredible impact our research has on children and families everyw...
12/23/2025

As the year comes to a close, weā€™re reflecting on the incredible impact our research has on children and families everywhere. When you support Children's Hospital of Philadelphia research, you become part of every breakthrough.

This December, help us end the year with momentum and hope. Your generosity fuels the high impact science that will shape the future of childrenā€™s health. Explore meaningful ways to give by visiting our Research Giving Page, and see how your support can bring healing and brighter tomorrows to families around the world: http://ms.spr.ly/6188tUyrO

CHOP investigators studied how rare inherited genetic variants contribute to the development of brain and spinal cord tu...
12/22/2025

CHOP investigators studied how rare inherited genetic variants contribute to the development of brain and spinal cord tumors. The findings, summarized in a new Snapshot Science blog, reveal insights into how hereditary genetic changes influence cancer risk and outcomes. Learn more about the study conducted with researchers at Childrenā€™s National Hospital: http://ms.spr.ly/6185toc7W

Designing clinical trials for vascular anomalies can be challenging due to different clinical presentations in patients ...
12/17/2025

Designing clinical trials for vascular anomalies can be challenging due to different clinical presentations in patients with the same genetic mutation. Now, Children's Hospital of Philadelphia researchers are bringing the rare disorder closer to clinical trial readiness by leading the creation of an expansive, multi-site patient registry and biorepository, Advancing Rare Disorders ā€“ Vascular mAlformation Research Network with CaNVAS (ARDVARC).

Learn more about how ARDVARC will transform how clinicians approach vascular anomalies using this robust new resource, in a new Cornerstone story: http://ms.spr.ly/6184tWaQK

12/17/2025

Our Center for Injury Research and Prevention at CHOP is welcoming applications for their unique summer internship! Applications for the Injury Science Research Experiences for Undergraduates (REU) will be accepted through Jan. 14, 2026. This 10-week paid internship sponsored by the National Science Foundation provides undergrads with mentorship and hands-on research experience in the fields of engineering, behavioral science, education, population science, and statistics. Learn more and apply now! http://ms.spr.ly/6181tWaoT

12/10/2025

šŸ—“ļø Mark your calendars: Applications for the Center for Injury Research and Prevention at CHOP's 2026 Injury Science Research Experiences for Undergraduates (REU) are open from Dec.15, 2025, to Jan. 16, 2026! This unique 10-week paid summer internship provides undergrads with mentorship from our experts and hands-on research experience in engineering, behavioral science, education, population science, and statistics. In this video, listen to 2025 interns Se'maj Griffin and Kasey Stammen answer the question: "Looking back, what stands out most from your internship experience at CHOP?"

Learn more: http://ms.spr.ly/6184tiVs0

Applications are now open for the Summer 2026 Advancing Representation in Research Administration (ARRA) Program at Chil...
12/04/2025

Applications are now open for the Summer 2026 Advancing Representation in Research Administration (ARRA) Program at Children's Hospital of Philadelphia Research Institute!

Undergraduate students from a wide variety of majors are invited to apply to gain practical training and a glimpse into what a career in research administration could look like.

Applications and references are due Jan. 25, 2026! Visit our website to learn about the program and register for our upcoming virtual information session. http://ms.spr.ly/6187td2SN

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3400 Civic Center Boulevard
Philadelphia, PA
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