BRAIN Publication-derived

SMA Patient Motor Neuron Organoids with SMN Restoration

Source Sances et al. · Cedars-Sinai Medical Center, Los Angeles, CA, USA · 10.1016/j.stemcr.2018.01.024

👤 Sances S, Ho R, Vatine G, West D, Laperle A, Meyer A, Workman M, Bhatt SM, Svendsen CN ⏱ 24000 min 🧫 Patient-Derived iPSC (SMA Type I)

Abstract

Sances et al. established SMA patient motor neuron organoids demonstrating SMN restoration and motor neuron rescue upon Nusinersen (ASO) and Risdiplam (small molecule) treatment.

Cell source

Patient-Derived iPSC (SMA Type I)

Application

SMA Disease Modeling

Full SOP

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Attribution

This SOP was authored by Organthis based on the published method in Sances et al.. The originating laboratory holds no rights in this SOP and has not endorsed it unless marked Verified.

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