MRNA THERAPY FOR ARGININOSUCCINATE SYNTHETASE DEFICIENCY

The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration in...

Ausführliche Beschreibung

Bibliographische Detailangaben

Hauptverfasser:	Derosa, Frank, Heartlein, Michael, Smith, Lianne
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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