Gene therapy for treating haemophilia: working to correct the body’s blood clotting “software”
VIB and Cellectis, announced that they are going to collaborate on research into new approaches to cure haemophilia. Thierry Vandendriessche and Marinee Chuah from the VIB Vesalius Research Center at Katholieke Universiteit Leuven are going to use meganucleases – tailor made enzymes engineered by Cellectis - to replace faulty blood clotting factor genes with functional copies.
Genetic therapy has long been considered a rational approach to cure haemophilia. However, one of the major setbacks to date was that new genetic material was inserted anywhere into the DNA, possibly disrupting useful genes, leading to unwanted side effects or the lack of expression of the gene of interest. Cellectis’ meganuclease technology enables accurate insertion of functional copies of the FVIII and FIX genes in a way that does not disturb the other functions of the targeted cell.
“Meganucleases have the potential to solve the random insertion issue,” commented Vandendriessche and Chuah from the VIB Vesalius Research Center at K.U. Leuven. “The technique allows the new genes to be delivered at a predetermined and safe location, with great precision. We have high hopes that one day, this research will lead to life-enhancing therapies for haemophiliacs.”
Gene therapy can be compared to reprogramming a cell’s software by uploading a corrective patch. The genetic code is the programming language, the mutated gene is the bug in the software and the meganucleases are the patch.
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Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
Topic world Gene therapy
Genetic diseases once considered untreatable are now at the center of innovative therapeutic approaches. Research and development of gene therapies in biotech and pharma aim to directly correct or replace defective or missing genes to combat disease at the molecular level. This revolutionary approach promises not only to treat symptoms, but to eliminate the cause of the disease itself.
