Engineers sharpen gene-editing tools to target cystic fibrosis Beyond cystic fibrosis, the refined base editor could help researchers tackle a wide range of genetic diseases caused by single-letter DNA changes.(Image: Bella Ciervo) Engineers sharpen gene-editing tools to target cystic fibrosis Researchers at Penn Engineering have developed a modified base-pair editor that offers improved accuracy and could help treat diseases like cystic fibrosis. 2 min. read
First CRISPR-based platform to pinpoint leukemia drivers Image: Alena Butusava via Getty Images First CRISPR-based platform to pinpoint leukemia drivers The new toolkit, developed by Penn Medicine and Children’s Hospital of Philadelphia, allows researchers to test potential cancer targets directly in patient leukemia cells. 2 min. read
Gene therapy ‘switch’ may offer non-addictive pain relief Image: nopparit via Getty Images Gene therapy ‘switch’ may offer non-addictive pain relief In a preclinical study, Penn Medicine researchers have uncovered a new gene therapy that targets only pain signals while leaving the rest of the brain untouched. 2 min. read
20 breakthroughs of 2025 The Polyhedral Structures Laboratory is housed at the Pennovation Center and brings together designers, engineers, and computer scientists to reimagine the built world. Using graphic statics, a method where forces are mapped as lines, they design forms that balance compression and tension. These result in structures that use far fewer materials while remaining strong and efficient.(Image: Eric Sucar) 20 breakthroughs of 2025 From ancient tombs and tiny robots to personalized gene editing and AI weather models, Penn’s 2025 research portfolio showed how curiosity—paired with collaboration—moves knowledge into impact and stretches across disciplines and continents. 5 min. read
Major gaps block genetics evaluation and testing for Black and low income patients Major gaps block genetics evaluation and testing for Black and low income patients Despite barriers to testing, a new study from Penn Medicine finds that Black patients benefit the most once they receive genetic testing results.
Baby KJ’s gene-editing treatment lands him on Nature’s top 10 list Penn In the News Philadelphia Inquirer Baby KJ’s gene-editing treatment lands him on Nature’s top 10 list The successful treatment by doctors at Penn Medicine and the Children’s Hospital of Philadelphia of a rare, life-threatening liver condition is spotlighted in a list of top scientific news for 2025. Startup recognized for milestones in cancer care Marco Ruella, associate professor of medicine at PSOM and hematologist-oncologist at Penn Medicine, collaborates with his colleagues in the Ruella Lab.nocred Startup recognized for milestones in cancer care During the Penn Center for Innovation’s 10th annual Celebration of Innovation, viTToria Biotherapeutics was presented with the Startup of the Year Award for its remarkable progress in the development of promising treatment of T-cell lymphoma. 4 min. read Early immune clues could help detect and prevent type 1 diabetes Image: Halfpoint Images via Getty Images Early immune clues could help detect and prevent type 1 diabetes Researchers from Penn Medicine have uncovered new clues in pancreas lymph nodes and the spleen that may stop the disease before insulin is lost forever. 2 min. read The baby whose life was saved by the first personalized CRISPR therapy Penn In the News Nature The baby whose life was saved by the first personalized CRISPR therapy Kiran Musunuru of the Perelman School of Medicine and Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia used a gene-editing therapy tailored to unique DNA sequences to treat a child with a rare genetic disorder. Identifying genes that keep cancer from spreading Histopathology tissue sections of the normal colon (left) showing highly organized cell-cell junctions (red) and proliferative zones (green) in colonic crypts. In contrast, a colon adenocarcinoma (right) is highly disorganized, with inconsistent cell junctions that can ultimately contribute to metastatic dissemination.(Image: Maggie Robertson) Identifying genes that keep cancer from spreading Using a novel approach, Penn Vet’s Chris Lengner and M. Andrés Blanco and colleagues have identified two genes that suppress colorectal cancer metastasis. 3 min. read Load More
Startup recognized for milestones in cancer care Marco Ruella, associate professor of medicine at PSOM and hematologist-oncologist at Penn Medicine, collaborates with his colleagues in the Ruella Lab.nocred Startup recognized for milestones in cancer care During the Penn Center for Innovation’s 10th annual Celebration of Innovation, viTToria Biotherapeutics was presented with the Startup of the Year Award for its remarkable progress in the development of promising treatment of T-cell lymphoma. 4 min. read
Early immune clues could help detect and prevent type 1 diabetes Image: Halfpoint Images via Getty Images Early immune clues could help detect and prevent type 1 diabetes Researchers from Penn Medicine have uncovered new clues in pancreas lymph nodes and the spleen that may stop the disease before insulin is lost forever. 2 min. read
The baby whose life was saved by the first personalized CRISPR therapy Penn In the News Nature The baby whose life was saved by the first personalized CRISPR therapy Kiran Musunuru of the Perelman School of Medicine and Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia used a gene-editing therapy tailored to unique DNA sequences to treat a child with a rare genetic disorder. Identifying genes that keep cancer from spreading Histopathology tissue sections of the normal colon (left) showing highly organized cell-cell junctions (red) and proliferative zones (green) in colonic crypts. In contrast, a colon adenocarcinoma (right) is highly disorganized, with inconsistent cell junctions that can ultimately contribute to metastatic dissemination.(Image: Maggie Robertson) Identifying genes that keep cancer from spreading Using a novel approach, Penn Vet’s Chris Lengner and M. Andrés Blanco and colleagues have identified two genes that suppress colorectal cancer metastasis. 3 min. read
Identifying genes that keep cancer from spreading Histopathology tissue sections of the normal colon (left) showing highly organized cell-cell junctions (red) and proliferative zones (green) in colonic crypts. In contrast, a colon adenocarcinoma (right) is highly disorganized, with inconsistent cell junctions that can ultimately contribute to metastatic dissemination.(Image: Maggie Robertson) Identifying genes that keep cancer from spreading Using a novel approach, Penn Vet’s Chris Lengner and M. Andrés Blanco and colleagues have identified two genes that suppress colorectal cancer metastasis. 3 min. read