Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.
Category Archives: Stories and News
Tommaso and his return to Normality
Tommaso was treated for ADA-SCID in February 2016 at the San Raffaele Telethon Institute in Milan (SR – Tiget).
A new ingredient to potentiate gene therapy in stem cells
A new study published by the research team led by Anna Kajaste-Rudnitski at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, shows that a naturally occurring compound significantly increases the efficiency of lentiviral vector-mediated gene transfer in blood stem cells.
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 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).
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.
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).
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
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
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.