11/15
Genetics
‘Mapping’ pancreatic cells at the development of Type 1 diabetes
Penn Medicine researchers examine of the underpinnings of the disease by creating a “map” to chart pancreatic islet cells over time.
How one researcher is helping us better understand the brain through epigenetics
Erica Korb, assistant professor of genetics, combines microscopy, bioinformatics, biochemistry, and behavioral testing to better understand gene expression and its impact on brain development.
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.
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.
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.
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.
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.
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.
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.
In the News
A Philly biotech got $60M from a TED initiative for AI in medicine
David Fajgenbaum of the Perelman School of Medicine helped found Every Cure, a biotechnology nonprofit that employs AI to help match existing treatments to new diseases.
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Study of gender-affirming care reveals immune system sex differences
Montserrat Anguera of the Perelman School of Medicine and the School of Veterinary Medicine comments on the work to comprehensively examine the impact of gender-affirming care on the immune system.
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A disease that makes children age rapidly gets closer to a cure
Kiran Musunuru of the Perelman School of Medicine says there’s no guarantee that gene editing which worked well in mice will also work with human patients.
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She’s fighting to stop the brain disease that killed her mother before it gets her
Kiran Musunuru of the Perelman School of Medicine comments on shutting off genetic signals in the brain to hold off diseases.
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FDA approves two sickle cell therapies, including first CRISPR medicine
Kiran Musunuru of the Perelman School of Medicine says that gene editing will be the biggest story of the century.
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More than 260,000 Penn Medicine patients have agreed to share their DNA for research, and the discoveries are just getting started
More than 260,000 people have signed up to participate in Penn Medicine BioBank, co-directed by Marilyn Ritchie and Dan Rader, which cross-references DNA with electronic health records to discover genetic variants of medical conditions.
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