Genetics | Penn Today

Five years of advancing innovative genetics researchers at Penn Medicine

A Penn master’s program celebrates its fifth graduating class of genetic counselors.

2 min. read

For the first time, a CRISPR drug treats a child’s unique mutation

Kiran Musunuru and Rebecca Ahrens-Nicklas of the Perelman School of Medicine led a team from Penn and the Children’s Hospital of Philadelphia that used gene editing to heal a baby with a rare genetic condition.

Baby healed in world’s first gene-editing therapy; Indian-origin doctor plays key role

Kiran Musunuru and Rebecca Ahrens-Nicklas of the Perelman School of Medicine led colleagues at Penn and the Children’s Hospital of Philadelphia to treat a baby with a rare genetic disorder using a customized gene-editing treatment.

This baby’s future was bleak. Then he became a medical first

A team from the Perelman School of Medicine and Children’s Hospital of Philadelphia led by Kiran Musunuru and Rebecca Ahrens-Nicklas has developed a customized CRISPR gene editing therapy that could be scaled to fit the needs of individual patients.

Kiran Musunuru and Rebecca Ahrens-Nicklas holding KJ in the hospital.

Livesaving personalized CRISPR editing therapy

A landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat a patient with a rare metabolic disease

5 min. read

First new subtype of Castleman disease discovered in 45 years

A new study co-authored by Penn Medicine’s David Fajgenbaum expands the spectrum of the rare disorder, which will help diagnose and treat patients caught between existing classification systems.

2 min. read

Genetic medicine can leave people with rare mutations behind. But there’s new hope

Kiran Musunuru of the Perelman School of Medicine says that there need to be a sufficiently large number of patients in a major market for a company to become interested in developing medications.

Mutated DNA restored to normal in gene therapy advance

Kiran Musunuru of the Perelman School of Medicine says that a new infusion offers the hope of precisely treating other genetic diseases by fixing mutations, an alternative to current gene therapies.

Penn-made AI tool matched a patient’s rare disease with a common inflammation drug. He’s been in remission for over two years

Every Cure, a nonprofit founded by David Fajgenbaum of the Perelman School of Medicine, has received $108 million in federal contracts and private financing to identify promising disease-medication matches and make recommendations for further clinical study.

Researchers create genetic map tied to kidney disease

The creation of the most complete map of more than 1,000 genes that influence kidney function could help experts diagnose and design targeted treatments for kidney disease.

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