What we do

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Who is doing it

Daphne Thijssen, Directeur Sanquin Bloodbank

Curing people indefinitely

“My dream? Within 20 years, patients with congenital blood diseases will be cured by gene therapy. They are then given their own repaired stem cells. That way, they are able to produce healthy blood themselves so that they are no longer dependent on regular blood transfusions.”

Who we do it for

Milka’s dream?

“Getting better with the help of gene therapy so that she herself will be able to make healthy blood cells again.”

“What if you are sick and only 0.001% of all blood donors are able to give you the right type of blood? That’s my reality. Because of my sickle cell disease I regularly need blood. I have developed so many antibodies against red blood cells that only a handful of blood donors worldwide can help me.”

Milka (20) has been receiving blood all her life and is for ever grateful to all donors. But imagine if Milka is again able to make healthy blood cells herself again, so that she is no longer dependent on the blood of donors?

Sanquin Research develops methods that can repair genetic defects in blood-forming stem cells. After returning these repaired stem cells, patients like Milka themselves would be able to make healthy blood cells.

What we need

With your donation to Sanquin Research Fund we can do further research into the complex technology needed to correct the genetic error in the DNA of these patients.

Repairing damaged cells at DNA level

Some patients suffer from a hereditary condition that seriously disrupts the production of blood cells. Sanquin researchers are developing technologies that can repair genetic defects in bloodforming stem cells. After returning their repaired stem cells, these patients will be able to make healthy blood on their own, so that they are no longer dependent on regular bloodtransfusions.
The technology needed to correct the genetic error in the DNA of these patients is complex. A lot of research is still needed, which we can start with the support of third parties. That would be fantastic. Because it would be more than groundbreaking if we can realize this dream.