abstract |
In particular, the present invention is a method for treating argininosuccinate synthase deficiency (ASD), wherein a composition comprising mRNA encoding argininosuccinate synthase (ASS1) is treated with at least one symptom or characteristic of ASD, It is provided for administration to a subject in need of treatment at an effective dose and dosing interval that reduces in intensity, severity, or frequency, or delays onset. In some embodiments, the mRNA is encapsulated within a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids. |