Cell-based gene therapy treatment cures hemophilia b in model

A novel, cell-based gene therapy approach was used to cure hemophilia B in a mouse model of the disease.
Hemophilia B, a blood-clotting disorder caused by defects in the gene encoding the protein-clotting factor IX (FIX) is considered to be an appropriate target for gene- and cell-based therapies due to its monogenic nature and broad therapeutic index. Investigators at the Salk Institute (La Jolla, CA, USA) used their FIX-deficient mouse model to analyse the use of cell therapy as a potential long-term cure for hemophilia B.At the first, they introduce that transplanted, cryopreserved, cadaveric human hepatocytes remained functional for more than a year and secreted FIX at therapeutic levels. Hepatocytes from different sources performed comparably in rectify the bleeding defect. Next, the investigators produce persuade pluripotent stem cells (iPSCs) from two hemophilia B patients and used the CRISPR/Cas9 gene-editing tool to correct the disease-causing mutations. These corrected iPSCs were coaxed into differentiating into hepatocyte-like cells (HLCs) and were then transplanted into hemophilic mice. Now investigator said Cell Reports tell that the transplanted iPSC-HLCs produced enough FIX to enable the mice to form normal blood clots and that the cells continued to survive and produce the FIX for at least nine to 12 months.
“The appeal of a cell-based approach is that you minimize the number of treatments that a patient needs,” said Dr Suvasini Ramaswamy, “Rather than constant injections, you can do this in one shot.”
Original Link:https://www.biotechdaily.com/genomics-proteomics/articles/294773535/cell-based-gene-therapy-treatment-cures-hemophilia-b-in-model.html