COMPOSITIONS AND METHODS FOR THE TREATMENT OF AADC DEFICIENCY

The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment, prophylaxis, palliation or amelioration of diseases, conditions and/or symptoms resulting from AADC deficiency and related inborn errors of neur...

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Bibliographische Detailangaben
Hauptverfasser:	Ravina, Bernard, Kotin, Robert, Kells, Adrian Philip
Format:	Patent
Sprache:	eng
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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Beschreibung
Zusammenfassung:	The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment, prophylaxis, palliation or amelioration of diseases, conditions and/or symptoms resulting from AADC deficiency and related inborn errors of neurotransmitter metabolism.