CPS-1 deficiency: Infant treated with gene therapy for the first time:
For the first time, an infant with the rare metabolic disorder CPS1 deficiency has been ...
ptaheute.de/aktuelles/2025/06/05/cps-1-mangel-saeugling-erstmals-mit-gentherapie-behandelt
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genetic modification of a patient's cells to produce a therapeutic effect
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