An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

Dopamine deficiency occurs when levels of the neurotransmitter dopamine are low, affecting movement, emotions, sleep, and ...