Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord

The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

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Bibliographische Detailangaben
Hauptverfasser: HOU, Jinzhao, PATZKE, Holger, CHEN, Qingmin, SAH, Dinah Wen-Yee, CARROLL SOPER, Jenna, HERSCH, Steven M
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.