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Our Research
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Overview
Research Question
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​How can we apply prime editing technology to allow for the suppression of the mutant HTT allele in patient-derived fibroblasts cells and reprogram them into induced pluripotent stem cells?
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Research Aims
Impact
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​Aim 1: To achieve selective insertion of stop codons into mutant allele in patient derived fibroblast cells bearing different SNPs.
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Aim 2: Induce selected cells into iPSCs and differentiate them to neurons.
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Methodology
Methodology
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We plan to use mesenchymal stem cells derived from bone marrow, as they have noted success in transplantation. These cells have the ability to differentiate into neural stem cells (NSC), which have demonstrated success coupled with CRISPR-Cas9.
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