In Vitro Validation of Antisense Oligoncleotide Targeting of KCNQ2-p.H228R

Project: Research project

Description

This proposal outlines in vitro experiments designed to demonstrate efficacy of antisense oligonucleotides (ASO) to target KCNQ2-p.H228R in human induced pluripotent stem cell (hiPSC)-derived cortical excitatory neurons using a combination of molecular biological and electrophysiological approaches.
StatusActive
Effective start/end date8/1/1912/31/19

Funding

  • Lydian Accelerator (Agmt. 7/7/2019)

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Induced Pluripotent Stem Cells
Antisense Oligonucleotides
Neurons
In Vitro Techniques