Genetics

Parents of baby saved by new gene editing therapy open up about his health journey

A team of researchers at CHOP and the Perelman School of Medicine developed a personalized gene-editing treatment for a baby’s rare disorder, using CRISPR to alter his DNA.

Determining the cause of cryopreservation fertility failures

A new study from Penn’s School of Veterinary Medicine on germ cell gene regulatory networks offers a holistic understanding of complex male germ cell differentiation in meiosis and spermatogenesis.

2 min. read

World-first CRISPR therapy could ‘transform’ treatment for rare genetic diseases, but key challenges lie ahead

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.

A search for new antibiotics in ancient DNA

César de la Fuente of the Perelman School of Medicine, School of Engineering and Applied Science, and School of Arts & Sciences and colleagues are probing genetic remnants of Neanderthals and extinct animals to find new antibiotics.

My patient with a rare condition should have formed a ‘second skeleton.’ Why didn’t he?

Frederick Kaplan of the Perelman School of Medicine shares his experience treating a patient with an unusual case of FOP, a rare genetic condition that causes the growth of a “second skeleton.”

Scientists edited genes inside a living person for the first time—and saved his life

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

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.

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