Oosterveen et al
The development of therapies to treat patients with neuronal indications is currently hampered by the use of animal models as less than 10% of findings derived from these preclinical models can be translated to humans. Patient-derived induced pluripotent stem cells (iPSCs) offer the possibility to generate in vitro systems to model neurological diseases that can recapitulate relevant human disease phenotypes. However, conventional human iPSC (hiPSC) differentiation protocols are often lengthy, inconsistent, and difficult to scale.
More importantly, the lack of genetically matched controls for patient-derived models further complicates the investigation of disease-relevant phenotype and study of molecular mechanisms underlying neurodegeneration. To overcome these problems, we developed a proprietary gene targeting strategy (opti-ox) that enables highly controlled expression of transcription factors to rapidly reprogram hiPSCs into pure somatic cell types in a scalable manner. Combined with CRISPR/Cas9-mediated genetic engineering enabled us to introduce specific mutations in these hiPSC-lines and create isogenic disease
will improve screen specificity and accelerate drug development
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