Through
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Genetics
Multidisciplinary team to develop stem cell-based approaches to restore vision
Multidisciplinary team to develop stem cell-based approaches to restore vision
Gene therapies have had success in treating blindness but can’t save areas of the retina where cells have already died. In a new effort, School of Veterinary Medicine scientists John Wolfe and William Beltran will attempt to develop a stem-cell-based approach that restores vision.
A study in prenatal gene editing with DNA in utero
A study in prenatal gene editing with DNA in utero
A Penn Medicine and CHOP team shows the first example of using base-editing tools to treat a disease in animal models in utero.
The next frontier of precision medicine: Parkinson’s disease
The next frontier of precision medicine: Parkinson’s disease
The Molecular Integration in Neurological Diagnosis (MIND) Initiative is working to understand the nature of Parkinson's disease with molecular-level accuracy, so doctors can treat the root cause.
Knockdown and replace: A gene therapy twofer to treat blindness
Knockdown and replace: A gene therapy twofer to treat blindness
More than 150 different mutations in the light-sensing molecule rhodopsin can cause retinitis pigmentosa, characterized by a progressive loss of night and peripheral vision, and a team of researchers have developed a treatment for the condition. Successful results in dogs set the stage for testing in humans.
Study finds mutation driving deadly brain tumors, and a potential remedy
Study finds mutation driving deadly brain tumors, and a potential remedy
Researchers at Penn and the Ludwig Institute for Cancer Research discovered that a mutation in the epidermal growth receptor gene indicates a severe glioblastoma, and targeting this particular gene may further treatment.
Correcting a blind spot
Correcting a blind spot
A groundbreaking genetic study seeks to transform the prevention and treatment of glaucoma while reversing historical racial disparities in who suffers from the disease, and who benefits from such research.
Could a new blood test predict preterm labor?
Could a new blood test predict preterm labor?
Research that included Medicine’s Michal Elovitz reveals that a non-invasive blood test might have the ability to determine a baby’s gestational age in utero and predict preterm delivery.
The varying skin colors of Africa: Light, dark, and all in between
The varying skin colors of Africa: Light, dark, and all in between
A team of geneticists led by Sarah Tishkoff, a Penn Integrates Knowledge Professor, has shown that there is a huge amount of variation of skin color within Africa, ranging from skin as light as some Asians to the darkest skin on a global level.
Race has a place in human genetics research, philosopher argues
Race has a place in human genetics research, philosopher argues
New research out of the philosophy department argues that certain racial classifications have utility in medical genetics, particularly when considering those classifications as ancestry groups.
Genetic variant largely found in patients of African descent increases risk for heart failure
Genetic variant largely found in patients of African descent increases risk for heart failure
A new study, led by Penn Medicine, reveals that this genetic cause of heart failure, which is now treatable, is significantly underdiagnosed.
In the News
The Washington Post
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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Philadelphia Inquirer
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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Philadelphia Inquirer
Jim Wilson has two new promising gene therapy studies, but says investment in the cutting-edge field has ‘gotten worse’
Jim Wilson of the Perelman School of Medicine has published two new studies supporting the promise of cutting-edge gene therapy, finding evidence that the genetic treatments can be beneficial for years without raising the risk of cancer.
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Smithsonian Magazine
Famed 5,300-year-old Alps Iceman was a balding middle-aged man with dark skin and eyes
Iain Mathieson of the Perelman School of Medicine says that the Iceman genome was one of the first ancient human genomes ever published.
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WHYY (Philadelphia)
Philly gene therapy companies tap into city’s workforce training program for lab techs
Penn Medicine is noted for spearheading gene therapy for cancer treatment, being the first in the nation to use a gene editing tool to combat cancer in 2019.
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Philadelphia Inquirer
These eight diseases are so rare that drug firms haven’t tried treating them with gene therapy. A $97 million program aims to help
Gene therapy for a rare form of blindness will be tested at Penn Medicine while gene therapy for a condition that causes skeletal deformities and seizures will be tested at the Children’s Hospital of Philadelphia.
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