Penn and CHOP Researchers Awarded $14 Million NIH Grant for Gene-Editing Research for Rare Metabolic Diseases
A $14 million grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children’s Hospital of Philadelphia (CHOP). The research will focus specifically on developing therapies for urea cycle disorders, which impact roughly 1 in every 35,000 children. Using a form of CRISPR technology, the ultimate vision of the four-year grant is to create a platform for rapid development of personalized gene-editing therapies for a wide range of rare genetic disorders.
The grant, funded by the National Institutes of Health through its Somatic Cell Genome Editing Program (SCGE), will support research to further advance prime editing, a new and more versatile form of CRISPR technology. Unlike previous gene-editing methods, prime editing allows precise changes to the genome, correcting any genetic mutation rather than just swapping out individual chemical bases of DNA. This technology holds the promise of personalized treatments for patients with rare metabolic diseases such as type I citrullinemia, ASA lyase deficiency, and CPS1 deficiency. These life-threatening conditions, characterized by the body’s inability to fully break down proteins, often lead to toxic ammonia buildup, causing brain damage, coma, or even death if untreated.
Despite previous attempts to treat these diseases through gene therapy, success has been limited due to immune responses to current therapies. Prime editing could change this by enabling permanent genetic corrections. “With this technology, we hope to not just manage symptoms, but offer a durable, potentially lifelong cure for these children,” said Kiran Musunuru, a professor of Cardiovascular Medicine and Director of Penn Cardiovascular Institute’s Genetic and Epigenetic Origins of Disease Program.
“We’re not just focusing on one specific disease,” said Rebecca Ahrens-Nicklas, an attending physician with the Metabolic Disease Program and the Division of Human Genetics at CHOP. “We’re focusing on the patient in front of us, whatever variant they have. This approach enables us to treat a wider array of patients who’ve previously had no options.”
The SCGE program is designed to address diseases caused by genetic changes. During its first phase (2018-2023), the program developed tools to perform genome editing in somatic cells which are non-reproductive cells in the body. Now in its second phase, SCGE seeks to bring genome-editing therapies from the lab to the clinic.
The team, which has previously received funding from the SCGE, aims to begin clinical trials within the next four years, marking an exciting new chapter in the field of precision medicine.
From the President and Provost: A Message to the Penn Community
October 21, 2024
To the Penn Community:
We write today regarding the 2024 Higher Education Sexual Misconduct and Awareness Survey. Along with nine other institutions, Penn engaged with this survey—as we did in 2015 and 2019—as a critical part of our longstanding commitment to protect and support students.
The goal of this survey was to gather information from our undergraduate, graduate, and professional students about sexual misconduct on campus, assess our education and prevention strategies, and guide our efforts moving forward. We thank the more than 7,300 Penn students who responded this year, updating this important data about students’ experiences.
With this year’s results, we note that prevalence rates have declined since 2019. However, we are deeply disturbed by the fact that high rates of sexual misconduct continue. Even one instance is too many. Sexual harassment and violence cross all lines of identity, ability, and background and disproportionately affect the marginalized and vulnerable among us.
In recent years, Penn has done much in collaboration with key campus partners to combat such violence and educate our community since 2019, notably:
- Invested in new staff positions in Penn Violence Prevention, the Office of the Associate Vice President of Equity and Title IX Officer, Restorative Practices@Penn, and Special Services in the Division of Public Safety.
- Enhanced programming for incoming students during New Student Orientation.
- Expanded partnerships and workshops across campus to provide immediate support for well-being as well as ongoing education for awareness and prevention.
- And increased student leadership, including the outreach and advocacy of the Penn Anti-Violence Educators (PAVE) and Abuse and Sexual Assault Prevention (ASAP), as well as establishing a Student Advisory Board for Penn Violence Prevention.
Despite these important steps forward, this year’s survey results show that we still have much to do to achieve our shared goal of eliminating sexual misconduct on our campus.
While there is much that these offices and interventions can do to mitigate sexual misconduct, this is an issue that requires our entire community to address. We urge all members of the Penn community to read the survey results, which can be found at https://ira.upenn.edu/surveys-penn-community/campus-climate-survey-sexual-assault-and-sexual-misconduct. Institutional initiatives and resources are crucial, and we rightly concentrate on healing and justice. But our goal must be prevention through education and intervention. We need every member of campus to join in. We strive to be a campus where all are safe and can thrive. So, it is incumbent upon all of us to partner toward prevention.
We also want to remind anyone who has experienced sexual misconduct—or knows someone who has experienced it—of the confidential resources available on campus:
- African-American Resource Center (resource for students, staff or faculty)
- Office of the Chaplain (resource for students, staff, faculty or visitors)
- Employee Assistance Program (resource for staff or faculty)
- Lesbian Gay Bisexual Transgender Center (resource for students, staff or faculty)
- Office of the Ombuds (resource for students, staff or faculty)
- Penn Violence Prevention (resource for students)
- Penn Women’s Center (resource for students, staff or faculty)
- Special Services Department, Division of Public Safety (resource for students, staff, faculty or visitors)
- Student Health and Counseling, including its Sexual Trauma Treatment Outreach and Prevention team also known as STTOP (resource for students)
- Wellness at Penn (resource for students)
Penn’s HELP Line is also available every day of the year, 24 hours a day at (215) 898-HELP (or 215-898-4357) to assist members of our community who are seeking help in navigating campus resources to support health and well-being.
Sexual misconduct is utterly unacceptable. We must and we will redouble our efforts and commitment toward prevention, awareness, and education. We are collectively responsible for one another and for building the campus environment we all want: one where every individual can learn and thrive free from violence or fear.
—J. Larry Jameson, Interim President
—John L. Jackson, Jr., Provost
Michael Mann: Penn’s Inaugural Vice Provost for Climate Science, Policy, and Action
Michael Mann has been named the University of Pennsylvania’s inaugural vice provost for climate science, policy, and action, effective November 1.
Dr. Mann is Presidential Distinguished Professor in the department of Earth and environmental science and director of the Penn Center for Science, Sustainability, and the Media.
In making the announcement, Provost John L. Jackson Jr. called Dr. Mann “one of the world’s leading experts in climate change and sustainability. We are deeply grateful to him for deciding to become a candidate and withdraw as a member of the consultative committee before the start of the search process and then for taking on this role at a pivotal time for global climate action.
“As vice provost, he will continue his essential work while partnering across campus to bring together the wide range of work already being done at Penn, leading innovations and catalyzing new collaborations.”
Dr. Mann is a globally renowned scholar of climate science whose many honors include the Tyler Prize for Environmental Achievement, Hans Oeschger Medal of the European Geosciences Union, Friend of the Planet Award from the National Center for Science Education, and John Scott Award from the City of Philadelphia. Elected to the Royal Society in 2024 and the National Academy of Sciences in 2020, he has been named one of the world’s most influential people in climate policy, one of the 10 most influential earth scientists, one of the top influencers in sustainability, and one of the 50 scientists who are changing the way we see the world.
Dr. Mann is an author and/or editor of six award-winning books and hundreds of publications across popular and scholarly media, including most recently Our Fragile Moment: How Lessons from Earth’s Past Can Help Us Survive the Climate Crisis (Public Affairs/Hachette, 2023), named one of the best books of the year by Financial Times; the widely acclaimed and influential The New Climate War: The Fight to Take Back Our Planet (Public Affairs/ Hachette, 2021), named one of the 20 Best Sustainability Books of All Time and to numerous other best books lists; and The Hockey Stick and the Climate Wars (Columbia University Press, 2012), based on his landmark contributions to the 2001 report of the United Nations Intergovernmental Panel on Climate Change, which included the now-famous “hockey stick” chart documenting the rise in global temperatures during the past thousand years.
Dr. Mann came to Penn in 2022 from Penn State University, where he was Distinguished Professor of Atmospheric Science and director of the Earth System Science Center. He taught at Penn State from 2005 to 2022, following earlier positions at the University of Virginia and University of Massachusetts and an Alexander Hollaender Distinguished Postdoctoral Research Fellowship from the Department of Energy. He received a Ph.D. in geology and geophysics and an M.S. in physics from Yale University and an A.B. in applied math and physics from the University of California, Berkeley.
“I thank Provost Jackson, Deputy Provost Beth Winkelstein, and the members of the consultative committee,” Dr. Mann said. “I couldn’t be more honored to help lead Penn forward in its mission to address the defining challenge of our time. We have all the key pieces in place, across our 12 schools, to lead on every aspect of the climate crisis—from the fundamental science to the impacts, solutions, and communication challenges—while exploring the ethical, sociological, and political dimensions of this predicament. In doing so, we honor the legacy of our founder, Benjamin Franklin—a statesman, a scholar, a scientist, and an environmentalist—as we proudly seek to make a better world. I look forward to the progress we will make together in the months and years to come.”
Penn Nursing Awarded $3.3 Million NIH Grant for Parents ASSIST Intervention for LGBTQ+ Families
The University of Pennsylvania School of Nursing has been awarded a $3.3 million grant from the National Institutes of Health (NIH) to develop and evaluate a new intervention designed to improve communication between parents and their gay or bisexual adolescent sons.
“Parents ASSIST: Intervention to Improve Parent Communication about Sexuality with Sexual Minority Male Adolescents,” is a five-year study led by principal investigator Dalmacio Dennis Flores, the Class of 1942 Endowed Term Chair; associate professor of nursing in the department of family and community health; and a senior fellow in Leonard Davis Institute of Health Economics. The study will focus on Gen Z youth (born between 1997-2012), who are coming out at earlier ages than previous generations. The intervention, known as Parents ASSIST, will provide parents with communication skills and LGBTQ+-specific health information.
“We are thrilled to receive this support from the NIH to address a critical need for families with adolescent sons who happen to have same-sex attractions, behaviors or identities,” said Dr. Flores. “This study will help us address the lack of evidence-based interventions to support parents helping their sons navigate adolescence. The investigation will involve a randomized controlled trial with a sample of 476 parent-adolescent dyads from across the country. Parents will be randomly assigned to either the Parents ASSIST intervention or a comparison group.
The researchers will assess the impact of the intervention on parent-adolescent sexuality communication, mental health outcomes for both parents and youth, parent-adolescent health behaviors, and family functioning. “We believe that this intervention can help families navigate the challenges associated with coming out and create a more supportive and inclusive environment for gay or bisexual male youth,” said Dr. Flores. “By improving communication and providing parents with the information they need, we can help to reduce disparities in mental health and other outcomes among this youth population.”
Dr. Flores’ co-investigators include Penn Nursing dean Antonia Villarruel and José A. Bauermeister, the Albert M. Greenfield Professor of Human Relations, and founding director of Eidos LGBTQ+ Health Initiative.
Sara Jaffee: Class of 1965 Term Professor of Psychology
Sara Jaffee, chair of the department of psychology in the School of Arts & Sciences, has been named a Class of 1965 Term Professor of Psychology. Dr. Jaffee, a developmental psychopathologist, conducts research on children and families experiencing a range of adversities, including poverty, housing insecurity, and violence. She studies the impact of these environments on development across the lifecourse and is interested in factors that support resilience to adversity.
Dr. Jaffee also studies whether policies that target structural determinants of health promote positive outcomes for children and families. Her work combines longitudinal, epidemiological methods with experimental and quasi-experimental designs to better understand how risk and protective factors operate in children’s development. The Class of 1965 Term Chairs were established in 1990 in honor of their 25th Reunion. The Class of 1965 endowed a chair for each of the four undergraduate schools and one in honor of the College for Women.
I. Joseph Kroll: Robert I. Williams Endowed Term Professor of Physics and Astronomy in the School of Arts & Sciences
I. Joseph Kroll has been named the Robert I. Williams Endowed Term Professor of Physics and Astronomy in the School of Arts & Sciences. Dr. Kroll’s research is in accelerator-based experimental particle physics, and he has worked on the study of proton-proton collisions, proton-antiproton collisions, and electron-positron collisions. He is currently a member of the ATLAS experiment at the CERN Large Hadron Collider in Geneva. There, his group has played a leading role in the search and discovery of the Higgs boson and in searches for as-yet-undiscovered particles that may explain unanswered questions in the current standard model of particle physics.
Dr. Kroll is a fellow of the American Physical Society and a fellow of the American Association for the Advancement of Science. He is the co-recipient of the 2013 European Physical Society (EPS) High Energy and Particle Physics Prize for the discovery of the Higgs boson and the 2019 EPS High Energy and Particle Physics Prize for the discovery of the top quark.
Paul C. Williams, W’67, established the Robert I. Williams Endowed Term Chair in memory of his father. At Penn, Mr. Williams is an engaged volunteer leader and has served in a variety of leadership roles. He is an emeritus trustee of the University, an emeritus member of the Penn Arts & Sciences Board of Advisors, and a former president of Penn Alumni. In 2011, he earned the University’s Alumni Award of Merit. He also served on the former biology department advisory board. In addition to his volunteer engagement, Mr. Williams has been a generous supporter of faculty, students, capital projects, and programs across the University.
Simone White: Associate Faculty Director of the Kelly Writers House
Simone White, whose work as poet, teacher, art critic, theorist, and performer has achieved international recognition, has been named associate faculty director of the Kelly Writers House. She has been closely affiliated with the Writers House since she joined the Penn faculty in 2018 and now joins faculty director Al Filreis, Director Jessica Lowenthal, and the staff, faculty, and students at 3805 Locust Walk in this new role.
As associate faculty director, Dr. White will work with her colleagues at the house to help develop new program series, focusing especially on interarts programming, encouraging the evolution of events and projects that benefit from the convergence of music, visual arts, poetics, sound studies, theory, and performance. With Dr. Filreis, Dr. White has taught the Kelly Writers House Fellows seminar—an annual program featuring three eminent writers who make extended visits to the house and to the class—and will continue to plan the visits of upcoming Fellows. With Dr. Filreis and others, she will participate in planning for the future of the Kelly Writers House as this uniquely capacious open house for writers on Penn’s campus prepares to celebrate its first thirty years in May 2026.
“Simone has already been an active and much admired member of the Writers House community,” wrote Dr. Filreis. “Teaching the Writers House Fellows seminar with her has been a dream. She is a truly great poet, has both natural and deeply learned instincts as a project- and event-maker, and leads in the most collaborative and conscientious way.”
Dr. White is the author of Warring (forthcoming from Duke University Press), or, on being the other woman (Duke University Press, 2022), Dear Angel of Death (Ugly Duckling Presse, 2018), Of Being Dispersed (Futurepoem, 2016), and House Envy of All the World (Factory School, 2010), the poetry chapbook, Unrest (Ugly Duckling Presse, 2013), and the collaborative poem/painting chapbook, Dolly (with Kim Thomas) (Q Ave, 2008). Her poetry and prose have been featured in Artforum, e-flux, Harper’s Magazine, BOMB Magazine, Chicago Review, Poetry, and The New York Times, among other publications. Her honors include the 2023 Dorothea Tanning Award from the Foundation for Contemporary Arts, a 2021 Creative Capital Award, and a 2017 Whiting Award in Poetry. A graduate of Wesleyan University, Dr. White holds a JD from Harvard Law School, an MFA from the New School, and a PhD in English from CUNY Graduate Center. She was raised in the Mt. Airy section of Philadelphia, about which she has written compellingly especially in her most recent poems.
“It’s such an honor to help steward this completely singular institution,” noted Dr. White. “The Writers House has a legacy of celebrating literature as a form of gathering together, an ethic I share as a teacher and a poet. I look forward to working with Al, Jessica, and the staff of Kelly Writers House to share our joy in studying and making literary art with students, the wider Penn and Philadelphia communities, and visitors from all over the world.”
Since joining Penn’s English department, Dr. White has taught graduate and undergraduate literature courses on Writing about Music, What Is Poetics?, W.E.B. DuBois, and creative writing workshops on Poetry Writing and Experimental Writing. She currently holds the rank of associate professor. She has been a member of the Kelly Writers House Advisory Board since 2020.
Launch of the Penn Advanced Research Computing Center
Provost John L. Jackson, Jr. and Senior Vice Provost for Research Dawn Bonnell announce the launch of the Penn Advanced Research Computing Center (PARCC), a groundbreaking initiative aligned with the goals of the University’s new strategic framework, In Principle and Practice, to lead on the great challenges of our time by harnessing interdisciplinary innovation to achieve the most impactful research outcomes.
PARCC, which will be available to researchers in spring 2025, will provide a central platform for computational and data-driven research across campus, including hardware, software, system support, and research consulting services. It will enhance power, capacity, and speed, with a high-performance computing data cluster housed at a regional colocation data center and access to additional GPUs and CPUs built on shared high-speed storage and networking infrastructure that will double current capacity at Penn.
PARCC will offer unparalleled support to researchers across all Penn schools and empower Penn’s research community to remain at the forefront of technological innovation. Its University-wide support and resources will provide the flexibility and economies of scale essential to achieve breakthroughs in data-driven and AI research. Over time, it will enable faculty and researchers to explore the best and most efficient research computing practices, including on-premises and cloud-based solutions, and provide additional resources such as data science consulting, enhanced HPC services, and increased capacity based on the evolving needs and growth of our computational research community. Researchers interested in PARCC can contact their school’s IT team and visit the new PARCC website for more details and to sign up for periodic updates that will include deadlines, pricing options, and additional technical specifications on the PARCC buildout.
All members of the Penn community are encouraged to visit the new PARCC website to learn more and to sign up for updates.