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Injection Improves Vision in a Form of Childhood Blindness
December 17, 2018
A new treatment for patients with a form of congenital retinal blindness has shown success in improving vision, according to results published today in Nature Medicine led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.
Genetics
Penn Medicine to Partner with the Allen Institute for Immunology
December 13, 2018
Penn Medicine will join four other leading clinical research organizations to understand the dynamic balancing act of the human immune system, how it senses friend from foe and what goes wrong during illness.
Genetics
Future Fertility: Giving Hope to Men Who Received Childhood Cancer Treatment
October 22, 2018
Researchers have discovered a way to grow human stem cells destined to become mature sperm in an effort to provide fertility options later in life to males who are diagnosed with cancer and undergo chemotherapy and radiation as children.
Genetics
Making Gene Therapy Delivery Safer and More Efficient
October 18, 2018
Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function, found researchers from the Perelman School of Medicine at the University of Pennsylvania in a study published in Molecular Therapy.
Genetics
There’s a Better Way to Decipher DNA’s Epigenetic Code to Identify Disease
October 08, 2018
A new method for sequencing the chemical groups attached to the surface of DNA is paving the way for better detection of cancer and other diseases, according to research from the Perelman School of Medicine at the University of Pennsylvania published today in Nature Biotechnology.
Genetics
Guided by CRISPR, Prenatal Gene Editing Shows Proof-of-Concept in Treating Congenital Disease before Birth
October 08, 2018
For the first time, scientists have performed prenatal gene editing to prevent a lethal metabolic disorder in laboratory animals, offering the potential to treat human congenital diseases before birth.
Genetics
Penn Medicine’s Orphan Disease Center Announces Three New Programs of Excellence
October 08, 2018
The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established three new Programs of Excellence that will focus on innovative gene therapies to treat rare immune, lymph node, and growth disorders.
Genetics
Knockdown and Replace: A Gene Therapy Twofer to Treat Blindness
August 20, 2018
New findings from a team led by University of Pennsylvania vision scientists, who have in the past taken gene therapies into clinical trials, are proving successful, this time treating a form of retinitis pigmentosa, a disease that progressively robs people of their night and peripheral vision before blindness develops.
Genetics
Genome Editing Reduces Cholesterol in Large Animal Model, Laying the Groundwork for In-Human Trials
July 09, 2018
Using genome editing to inactivate a protein called PCSK9 effectively reduces cholesterol levels in rhesus macaques, a species of monkey, according to researchers from the Perelman School of Medicine at the University of Pennsylvania.
Genetics
Penn Medicine Biochemist Receives Major Award for Research on Epigenetic Protein Modifications via Mass Spectrometry
June 15, 2018
Benjamin A.Garcia, PhD, an expert in quantitative proteomics and Presidential Professor of Biochemistry and Biophysics in the Perelman School of Medicine at the University of Pennsylvania, has been awarded the Biemann Medal by the American Society for Mass Spectrometry (ASMS).
Genetics
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