Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 <i>α</i> 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

Abstract excerpt

A phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 (rAAV2) alpha1-antitrypsin (AAT) vector was performed in 12 AAT-deficient adults, 10 of whom were male. All subjects were either homozygous for the most common AAT mutation (a missense mutation designated PI*Z) or compound heterozygous for PI*Z and another mutation known to cause disease. There were four dose cohorts,...

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