A Biorepository of Angelman syndrome IPSC Lines and Brain Organoids | $1,068,455

The patient iPSC derived neurons, and brain organoids (aka mini-brains), become essential tools or systems to test various approaches in human therapeutics, especially for sequence specific neurogenetic disorders (e.g. ASO and CRISPR/Cas9 based therapies, as well as other potential molecular treatments for AS). The major goal of this proposal is to create a high-quality biorepository of all genotypes using human AS patient derived IPSC lines and brain organoids that could be shared with the AS research community both in academic and industrial institutes in order to be able to test various therapeutic compounds and understand effect and tolerability with those compounds.

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