Genetics | Penn Today

Changing the identity of cancer cells to eliminate them

A team led by the School of Veterinary Medicine’s M. Andrés Blanco has uncovered a new target for treating certain blood cancers that works by removing an obstacle to their maturation.

Novel gene therapy for hemophilia A

The multicenter study, led by researchers at the Perelman School of Medicine and Children’s Hospital of Philadelphia, showed improved and sustained production of a needed clotting factor and reduced bleeding events.

DNA helix with a piece being removed by a surgeon’s glove with scissors.

New gene-editing technique can ‘turn on’ enzymes that cause DNA base mutations

The novel Penn method of splitting specific mutator enzymes and then triggering them to reconstitute has been patented, and allows for new possibilities in biologic and therapeutic research, including cancer.

Visual representation of a DNA sequence.

Moving past conflation of race and genetics

Race is not genetic. Race is a social and political construct. However, the conflation of race and genetics is one way that racism persists in medicine and research.

Home DNA tests can lead to shock and trauma, but mental health resources are scarce

Dana Farengo Clark of the Abramson Cancer Center said many people come into the Center looking for support after taking a home DNA test that revealed their risk, as well as family members’ risk, for cancer. “A piece of the psychology of this is not only getting the result for yourself but then being the gatekeeper of that information,” she said.

A brain scan with highlighted genetic activity.

Genetic disease CDKL5 deficiency disorder could be treatable after childhood

The study finds that the CDKL5 gene, which plays an important role in proper brain development during childhood, is also active in adulthood, and that gene therapy may help adult patients.

diagram of gene therapy for the eye showing injection into vitreous

Novel gene therapy platform speeds search for ways to cure blindness

A newly developed single-cell RNA sequencing technique enables researchers to quickly identify an optimal vector for delivering therapeutic genetic material to treat vision disorders, and perhaps other genetic conditions.

Four success stories in gene therapy

Jean Bennett and Albert Maguire of the Perelman School of Medicine developed a gene therapy to treat blindness in patients with retinal dystrophy caused by mutations in the RPE65 gene. “These people can now do things they never could have dreamed of doing, and they’re more independent and enjoying life,” said Bennett.

National Academy of Medicine welcomes two new members from Penn

The Perelman School of Medicine’s Marylyn D. Ritchie and PIK Professor Sarah A. Tishkoff are among 100 new members to be elected this year to the Academy, one of the highest honors in health and medicine.

Image showing body with shoulder, wrist, elbow, and hip joints in red indicating pain and inflammation

A two-pronged approach to keep rheumatoid arthritis in check

A new study led by George Hajishengallis of the School of Dental Medicine shows that the protein DEL-1 could reduce the painful inflammation of RA in an animal model.

Filters