WHEN MOM IS NOT ENOUGH

Gene therapy using hematopoietic stem cells has shown remarkable results in treating primary immunodeficiencies, rare genetic diseases. However, collecting these cells from younger children remains a significant challenge.

With your support, we aim to fund research focused on a gene therapy approach that involves the collection, genetic correction, and re-infusion of the patient’s own blood stem cells.

For very young children, traditional methods of harvesting hematopoietic stem cells can be technically difficult. The research we want to fund with your help is focused on studying stem cells that naturally circulate in the peripheral blood, proposing them as a new target for innovative therapies. Thanks to recent technological advances, these circulating hematopoietic stem cells could be genetically corrected directly within the patient, without the need for harvesting them.

This approach would allow us to:

• Intervene early, preventing the onset of the disease
• Reduce or eliminate the need for patient hospitalization during stem cell collection
• Eliminate transplant-related toxicity, as it would no longer be necessary

Overall, this project, led by the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, will lay the groundwork for the clinical application of circulating stem cells as a new cellular target for timely gene therapy in infants during the first months of life.

WHEN MOM ISN’T ENOUGH…
LaSpes, I AM RARE, BUT I EXIST.

With your help, we can make a difference!

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