We work everyday for:

Every life counts

Mostly overlooked by large public and industrial investments, orphans of research and drugs: rare genetic diseases taken individually are not statistically significant. But can we say the same for somebody’s life?

Why do we study genetic diseases?

Research can give real hope

By funding research we can provide answers and therapies to people who otherwise, in many cases, would not have any hope.

Learn more about our mission.

Latest news

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A new ingredient to potentiate gene therapy in stem cells
Gene therapy has reached significant clinical milestones but its application remains difficult for a number of disorders and cell types for which basic knowledge to develop more efficient protocols and strategies is still lacking. Now, a new study published in Cell Stem Cell* by the research team...
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Restore Health – Therapies that cure
On 18 May, the European Commission (EC) announced that the FET Flagship RESTORE, which was initiated by the Berlin-Brandenburg Center for Regenerative Therapies (BCRT), has been selected to enter the next phase of the FET Flagship competition. A successful FET Flagship will eventually receive ar...
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From epigenetics - new molecular switches to silence genes
It is possible to silence a desired gene by hiding it from the molecular machinery that has the task of reading it and expressing its function. This is the result of a research carried out at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) published this week in Cell. This techni...
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Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele
Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, and Fondazione Telethon and Ospedale San Raffaele, which operate a joint research collaboration known as the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) have entered into a scientific collaboration to research...