Step 1
A patient would receive a one-time I.V. administration of HMI-103 consisting of functional copies of the PAH gene with a liver-specific promoter surrounded by long DNA sequences homologous to a specific location in the genome (homology arms).
A patient would receive a one-time I.V. administration of HMI-103 consisting of functional copies of the PAH gene with a liver-specific promoter surrounded by long DNA sequences homologous to a specific location in the genome (homology arms).