Genome Editing of Blood Coagulation Factor VIII (F8) in Hemophilia A Patient-Derived iPS Cells

Item Number: 582
Industry: Therapeutic
Category: Cardiovascular / Haematology, Gene Therapy
Development Stage: Early Stage
Organisation Type: University
Country: South Korea
Patent: Korea, PCT
Business Opportunity: Out-Licensing, Technology Transfer, Collaboration R&D


• Modeling hemophilia in degenerated stem cells using the gene scissors  (TALEN)  

• First mutant gene correction using CRISPR / Cas9 in iPS cells of patients

• Corrected cells had no nonspecific mutations and we identified the validation through hemophilia mouse model

• Identify the possibility of cell therapy through self-transplantation

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