The Children's Hospital of Philadelphia Research Institute

Experts at Children's Hospital of Philadelphia and the University of Pennsylvania gathered for the CHOP-Penn Autism Research Symposium: Building the Future Together. The event highlighted the unique and strong partnership between the two institutions, as well as their immense potential to further improve how we diagnose and treat autism spectrum disorder across the lifespan.

“Imagine a future where we bring together the remarkable talent gathered here to solve challenges faced by autistic patients and families,” said Dr. Susan Furth, Executive Vice President and Chief Scientific Officer at CHOP Research Institute, at the symposium. “The future is bright for us as a community to make an enormous impact.”

Read our wrap-up of the May 9 CHOP-Penn Autism Research Symposium on Cornerstone now:
http://ms.spr.ly/6180Yvxz0

Our June Faculty Spotlight features Dr. Suzanne MacFarland, Division of Oncology at Children's Hospital of Philadelphia, who studies pediatric cancer predisposition. Dr. MacFarland is driven by the desire to help patients and their families understand why a cancer might have developed and identify future risks. In a new Cornerstone Q&A, learn more about her research, including a new grant to investigate the potential genetic causes of juvenile polyposis syndrome, a condition that can have an increased risk of gastrointestinal cancer later in life. http://ms.spr.ly/6184YqmB6

This National Gun Violence Awareness Day, we are proud to see Children's Hospital of Philadelphia staff in support of ending gun violence. At CHOP, our Center for Violence Prevention (CVP) leads a number of unique research projects and initiatives to prevent gun violence and its harmful impact on mental health. Learn more in our Where Discovery leads story: https://www.research.chop.edu/cornerstone-blog/informing-novel-interventions-for-gun-violence-prevention

Photo credit: Center for Injury Research and Prevention at CHOP

Children's Hospital of Philadelphia celebrated the naming of its newest research facility, the Morgan Center for Research and Innovation, with a beam signing event June 3. A transformational gift of $50 million from the Morgan family ushers in a new era of our hospital's 169-year-old history. Read highlights from the event on Cornerstone:
http://ms.spr.ly/6182YWof0

It’s out of the laboratory and into the woods for Drs. David Stokes and Arthur Lee in our first blog post of 2024. In this seasonal blog series, we share the hobbies and interests of Children's Hospital of Philadelphia Research Institute researchers and staff.

On campus, Dr. Stokes is the technical director of CHOP’s Biorepository Resource Center. Off campus, he’s a wildlife photographer with a passion for remote locations and the life they contain.

Meanwhile, Dr. Lee spends his workdays as a Nephrology postdoctoral fellow and his weekends hiking scenic locales in the company of 30 to 50 pounds of camera equipment.

Read more on Cornerstone: http://ms.spr.ly/6189YcmMW

Meet this year’s recipients of the Children's Hospital of Philadelphia Award for Excellence in Mentoring Research Trainees! This special honor recognizes members of our scientific community who go above and beyond to devote their time, energy, expertise, and empathy to the next generation of CHOP scientists. Congratulations to Dr. Amelia J. Eisch (Anesthesiology and Critical Care Medicine), Dr. Chén C. Kenyon (Pediatrics), and Dr. Frederick “Chris” Bennett (Child and Adolescent Psychiatry and Behavioral Sciences)!

Read all about these exceptional mentors in a new Cornerstone blog:
http://ms.spr.ly/6181YiCwF

Dr. Warren Frankenberger, a nurse scientist at Children's Hospital of Philadelphia, received grant support from the Eunice Kennedy Shriver National Institute of Child Health and Human Development to further his work in pediatric emergency care. Building on previous research through a major collaboration between the CHOP Center for Pediatric Nursing Research and the Arcus team at CHOP Research Institute, he aims to determine the accuracy of an emergency triage tool in the pediatric population. Learn more: http://ms.spr.ly/6189YiCXb

The U.S. Food and Drug Administration approval of a new gene therapy for hemophilia B could change the lives of many adult patients who currently rely on daily therapeutic injections to treat their disorder. But to understand the breadth of this breakthrough, we must go back to the beginning — when hematologist Dr. Katherine A. High was determined to improve existing therapies for hemophilia B in the 1980s.

On our Cornerstone blog, read about the journey that spans three decades of basic and clinical research, as Dr. High and her colleagues at Children's Hospital of Philadelphia and the University of Pennsylvania began writing the playbook for gene therapy research and development.

http://ms.spr.ly/6186YiWh0

The U.S. Food and Drug Administration approval of a new gene therapy for hemophilia B could change the lives of many patients over 18 who currently rely on daily therapeutic injections to treat their disorder. But to understand the breadth of this breakthrough, we must go back to the beginning — when Dr. Katherine A. High was determined to improve existing therapies for hemophilia B in the 1980s.

On our Cornerstone blog, read about the journey that spans three decades of basic and clinical research, as Dr. High and her colleagues at Children's Hospital of Philadelphia and the University of Pennsylvania began writing the playbook for gene therapy research and development.

http://ms.spr.ly/6184YiWCg

Today we capped off Research Staff Recognition Day at Children's Hospital of Philadelphia with an award luncheon for 25 nominees of the Outstanding Research Staff Award. These incredible research technicians, lab specialists, scientists, lab managers, and lab directors were nominated by CHOP faculty, postdoctoral fellows, physician fellows, and instructors for their exemplary support in the conduct of research and training.

Join us in congratulating the three winners: Hillary Kapa (nominated for Leadership in Research), Livia Sertori Finoti (nominated for Scientific Merit/Excellence in Research), and Joe Chan (nominated for Commitment to Training) as well as all the amazing nominees. A special thank you to our Office of Academic Training and Outreach Programs for their development, coordination, and organization of this appreciation event!

We're celebrating Children's Hospital of Philadelphia Research Lab Staff today! From now until 12PM, our Office of Academic Training and Outreach Programs (ATOP) will be in the Abramson Pediatric Research Center Lobby with free coffee and giveaways for all CHOP lab staff. Learn more about Research Staff Recognition Day and the full list of offerings: https://www.research.chop.edu/events/research-staff-recognition-day-may-29-2024

Congratulations to Dr. Scott Adzick, CHOP's Surgeon-in-Chief and founder and Director of The Center for Fetal Diagnosis and Treatment at CHOP, who was the recipient of the 2024 Robert E. Gross Award for Excellence in Pediatric Research and Achievement by the American Pediatric Surgical Association!

This award recognizes a seminal contribution by an individual who has made a major impact on pediatric surgery, whose innovative contributions have resulted in a significant change in how pediatric surgeons manage a particular problem, and the results have been demonstrated to be durable with time and widely accepted by pediatric surgeons as “standard practice." We couldn't be prouder of Dr. Adzick! Read more: http://ms.spr.ly/6184Ye0tM

CHOP’s Surgeon-in-Chief N. Scott Adzick Wins 2024 Robert E. Gross Award | Children's Hospital of Philadelphia The American Pediatric Surgical Association awarded Dr. N. Scott Adzick the 2024 Robert E. Gross Award for Excellence in Pediatric Research and Achievement.

A decade ago, Sarah McCague never imagined she would be managing a team of clinical research staff for one of Children's Hospital of Philadelphia's most innovative research teams: the Clinical In Vivo Gene Therapy (CIGT) group. Sarah has facilitated some of CHOP’s most impactful clinical studies, including trials for a gene therapy to treat an inherited form of blindness — the first gene therapy approved by the U.S. Food and Drug Administration for a genetic disease.

In a new Cornerstone story, Sarah describes the unique skills of CHOP’s gene therapy clinical research staff — roles that comes with as much reward as they do responsibility.

Read Sarah’s story now: http://ms.spr.ly/6187Yw0XL

Join us in recognizing our incredible clinical research staff at Children's Hospital of Philadelphia! On May 7, CHOP Research hosted the 11th annual Clinical Research Coordinator Research Excellence (CRC RE@CH) Awards and Recognition Event. The celebration featured a touching keynote speech from Faith Alunni, a CRC and former CHOP patient headed to med school, followed by a ceremony for the three recipients of the CRC RE@CH Award: Laura MacMullen (nominated by Dr. Marni Falk in the The Mitochondrial Medicine Center at CHOP), Ahtish Arputuhan (nominated by Dr. Andrea Kelly in the Division of Endocrinology), and Kristina Ziolowski (nominated by Dr. Sara DeMauro in the Division of Neonatology).

Read highlights from the special day on Cornerstone now: http://ms.spr.ly/6181YwLIN

Faith Alunni, a clinical research coordinator at Children's Hospital of Philadelphia who is headed to medical school this fall, knows the weight of her work with patients and families intimately.

Twenty-four years ago, Faith was born at CHOP joined at the chest and abdomen to twin sister, Hope. Prior to their birth, The Center for Fetal Diagnosis and Treatment at CHOP found that though the sisters had separate hearts, Hope needed the strength of her sister’s heart to survive. It was a harrowing time for Faith's family. Within seconds of the twins' separation, Hope passed away.

Hope's story, however, doesn't end there. Faith has carried her sister's spirit with her into a purposeful and passionate career working with patients, families, and principal investigators to drive research forward and change children's lives. After med school, Faith hopes to become a pediatric surgeon.

Clinical research staff like Faith are the heart and soul of CHOP Research, and on , we extend our gratitude for their professionalism, compassion, and hope in our breakthroughs.

Our researchers at Children's Hospital of Philadelphia shared an alternative conditioning regimen for treating beta thalassemia, a severe blood disorder, at the American Society of Gene & Cell Therapy’s 27th Annual meeting. Learn more about how this new model could someday significantly improve the lives of patients by avoiding severe side effects of chemotherapy or radiation: http://ms.spr.ly/6182YZYaA.

Happy 25th anniversary to Children's Hospital of Philadelphia Research Institute's first Center of Emphasis! 🎉 The Center for Injury Research and Prevention at CHOP (CIRP) celebrated Tuesday evening with its original founders, Drs. Flaura Winston and Dennis Durbin in attendance alongside CHOP leaders, investigators, collaborators, students, staff, and more.

In 1996, Drs. Winston and Durbin dreamed of raising the rigor of injury research as it applied to children. They brought together a team of scientists and clinicians who began building an evidence base to educate families, inform policies and regulations, influence laws, and make the world safer for children and adolescents.

Today, CIRP is an ambitious and multidisciplinary center with a broad range of focus areas from young driver safety to violence prevention to concussion and brain health, and so much more. Learn more about the future of CIRP at CHOP: http://ms.spr.ly/6185YktBH

“Asian American and Pacific Islander Heritage Month is a time for reflection, support, and celebration of the valuable contributions made by the AAPI community to the nation’s economic, technological, social, and political landscape while navigating significant systemic and cultural obstacles,” – Dr. Namrata Kumar.

This , read a new Q&A with our May featured research trainee, Dr. Kumar, a postdoc in Children's Hospital of Philadelphia's Weitzman Lab and a current co-chair of the CHOP Postdoctoral Alliance.

After earning an undergraduate degree at the University of Pune in India, Dr. Kumar completed her master’s degree at the University of Texas and doctorate at the University of Pittsburgh School of Medicine. In this Q&A, she discusses CHOP’s unique opportunities for professional growth, from mentoring to certificate programs to supporting colleagues through the CHOP PostdocAlliance.
http://ms.spr.ly/6180YX4wu

At Children's Hospital of Philadelphia Research Institute, Dr. Jennifer Lynch, a cardiac anesthesiologist and physicist, and her team are developing a unique noninvasive neuromonitoring research device to study the risk of neurologic injury in children with congenital heart disease (CHD).

Neurodevelopmental disabilities remain a significant morbidity among CHD survivors. In the “not so distant future,” Dr. Lynch envisions the lab’s advanced technologies will help clinicians monitor neurologic risk in cardiac operating rooms, catheterization labs, and critical care units. Learn more on Cornerstone: http://ms.spr.ly/6185YV63l

Cell and gene therapy experts from Children's Hospital of Philadelphia are at ! Here are three facts about the unique expertise of our Clinical Vector Core (CVC). Meet our CVC team at Booth #613:

▪️ The CVC brings over 20 years of experience in adeno-associated virus and lentivirus manufacturing.
▪️ The CVC is currently supporting 39 early phase clinical trials.
▪️ The CVC provides U.S. FDA, European Medicines Agency, and Healthcare Products Regulatory Agency compliant vector manufacturing for early phase clinical trials.

Ask our CVC team about these and other facts today until May 11. Not attending ASGCT this year? Learn more on our website: http://ms.spr.ly/6186YVRv0

This , read about Children's Hospital of Philadelphia nurse scientist Dr. Clifton Thornton, founding member of a unique collaboration that helps survivors succeed at school by combining expertise in nursing science, education, & psychology:
http://ms.spr.ly/6181YV6Ez

CHOP restored eyesight for two children with one injection. It’s one more step for gene editing using CRISPR. The 10-year-old and 14-year-old patients had their eyesight improved about 10- to 100-fold after a single injection to their retina.

Good morning to all those attending this week! Our Clinical Vector Core (CVC) team at Children's Hospital of Philadelphia looks forward to meeting you at Booth #613 and discussing how they can support your and work. Why should you stop by? The CVC's leadership and legacy in viral vector manufacturing includes:

▫️ Production of 177+ vector products for 39 unique clinical indications including vectors supporting the FDA's first-ever approved gene therapies tisagenlecleucel and voretigene neparvovec-rzyl.
▫️Support for early-phase studies with vector production that is affordable and completed within a short timeframe.
▫️ In-house expertise in clinical research and products at CHOP, a world-class institution with one of the highest number of ongoing cell and gene therapy trials today.

Learn more about the CVC: http://ms.spr.ly/6187YpPJt

Earlier this week, the next generation of pediatric researchers presented more than 100 abstracts at 2024 Children's Hospital of Philadelphia Research Poster Day with the opportunity to win a Poster Day Award. Join us in congratulating the 20 winners in the Patient-Oriented Research Track, judged by Dr. Allison E. Curry and the 20 winners in the Laboratory-Based Research Track, judged by Dr. Yuanquan Song.

A special congratulation also goes to the winners of the Science Slam Flash Talks, where selected poster presenters wrapped complex research into a three-minute, one-slide presentation. The Audience Choice Science Slam Winners were Veronica Bochenek and Evan Rosenberg!

Check out a full list of 2024 Poster Day award winners now:
http://ms.spr.ly/6187YRdFN

Members of our Omics & Big Data Initiative at Children's Hospital of Philadelphia are at Poster Day and Scientific Symposium (PDSS) this year to share the power of ! The Initiative's groundbreaking programs harness the power of omics data - from proteomics to genomics to epigenomics and more - to fuel precision medicine research and optimize patient care at CHOP. If you didn't get a chance to chat with the team today, they will be at PDSS tomorrow, May 1, in the Hub for Clinical Collaboration lobby. (PDSS is an in-person event open to all members of CHOP and the University of Pennsylvania.)

Pictured here are some of the stellar individuals working within the Omics Initiative: Elizabeth DeChene, senior manager of operations; Alexandra Heck, clinical research coordinator; Anna Melamed, clinical research coordinator; Kate Driesbaugh, CHOP Biobank manager; and Alex Gonzalez, senior manager of Translational Research Informatics Group (TRiG) in the Department of Biomedical and Health Informatics.

Can't make it to PDSS? Learn more about the programs launched by the Omics Initiative: http://ms.spr.ly/6183YOlj3

Today at Children's Hospital of Philadelphia Research Institute, our "Galaxy of Safety" professionals showed the CHOP Research community just how engaging can be. The Office of Research Safety (ORS) at CHOP launched its first in-person Safety Day since 2019, bringing back games, prizes, vendors, administrative booths and giveaways that revolved around the important themes of laboratory safety, fire safety, chemical safety, biological safety, shipping safety, and more. Without the passionate and proactive team at ORS - and the who practice safe science every day - our breakthroughs in children's health would not be possible.

Did you attend Research Safety Day today? Tag yourself and head over to our CHOP Research Instagram account to see more highlights from the fun-filled and educational day! http://ms.spr.ly/6185Y3YQU

Our April , Dr. Elizabeth McGovern, was inspired by her clinical work as an anesthesiologist and intensivist to research unique and novel approaches to improving skills in high-risk pediatric airway procedures like tracheal intubation.

“By developing these approaches for assessing and tracking procedural competency and developing targeted interventions, I hope we can improve skills for tracheal intubation and other high-risk procedures to better care for our patients,” Dr. McGovern said.

Read the full Q&A now: http://ms.spr.ly/6187YyR7S

A fascinating new study from Children's Hospital of Philadelphia and University of Pennsylvania researchers reveals the important role the immune system and the microbiome play in passing down non-genetically inherited phenotypes to impact future generations. The study not only expands how scientists understand the introduction of short-term phenotypic diversity without genetic-based mutations, but also helps to contextualize how past environments and experiences influence the state of human health. Learn more in a new Snapshot Science:
http://ms.spr.ly/6180Yyr6K