Restore Health – Therapies that cure

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.

From Telethon Foundation


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).

From Research


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, have entered into a scientific collaboration to research and develop genome edited hematopoietic stem cell (HSC) and T cell therapies.

From Telethon Foundation


Preliminary results reporting the safety and effectiveness of gene therapy in patients with metachromatic leukodystrophy published in The Lancet

The gene therapy developed in the laboratories of the San Raffaele Telethon Institute for Gene Therapy in Milan (SR-Tiget) remains effective as a potential early treatment of metachromatic leukodystrophy (MLD).

From Research


Strimvelis receives European marketing authorisation to treat very rare disease, ADA-SCID

GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID

From Telethon Foundation


GSK receives positive CHMP opinion in Europe for StrimvelisTM, the first gene therapy to treat very rare disease, ADA-SCID

GlaxoSmithKline (LSE/NYSE: GSK) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending marketing authorisation for Strimvelis

From Telethon Foundation


Gene therapy: a "molecular scalpel" corrects the genes of blood stem cells

A group of researchers at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan was able for the first time to rewrite the DNA of human blood stem cells by editing the genome.

From Research


Cardiomyopathies: a protein has been discovered that keeps the heart young

It's called Atrogin-1 and it's a protein that plays an important role in cardiac health: its task, as demonstrated by a study funded by Telethon, is to identify and dispose of the waste substances produced by cellular activity, which would be harmful to the heart.

From Research


Ipex syndrome: fresh hope for a cure from Telethon - San Raffaele researchers

From Telethon - San Raffaele research come new treatment prospects for a rare genetic diseases affecting the immune system, IPEX syndrome.

From Research

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