title: Gene-splicing protein to have orphan drug status.
mesh_terms: D005818:Genetic Engineering; D006801:Humans; D007880:Legislation, Drug; D014481:United States; D014486:United States Food and Drug Administration; D000515:alpha 1-Antitrypsin / Q000235:genetics* / Q000627:therapeutic use
chemical_list: D000515:alpha 1-Antitrypsin