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

Item Number: 590
Industry: Therapeutic
Category: Cardiovascular / Haematology, Gene Therapy
Development Stage: Early Stage
Organisation Type: University
Country: South Korea
Business Opportunity: Out-Licensing, Technology Transfer, Collaboration R&D


Using the gene scissors (TALEN) to model the world's first hemophilic disease in degenerated stem cells

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

  • Corrected cells had no nonspecific mutations / Identify the validation in hemophilia mouse model
  • Identify the possibility of cell therapy through self-transplantation

Features & Contact

Sign in and you'll be able to send enquiries about this item, view full details and save it