News
28.08.25
How Newborn Liver Cells Shape Growth and Future Treatments
A discovery at SR-Tiget reveals the key liver cells driving organ growth and advancing pediatric gene therapy
06.08.25
New 3D imaging tech revolutionizes lysosomal disease research
A new HTFC method allows live 3D lysosome imaging, with applications in Niemann-Pick C1 diagnosis and monitoring
30.07.25
Usher Syndrome: Promising Preliminary Results from Gene Therapy
An experimental gene therapy developed at the TIGEM has shown encouraging clinical results in patients with Usher syndrome type 1B.
17.06.25
TIGEM secures ERC grant for programmable gene‑circuit therapy
di Bernardo’s DIMERCIRCUITS integrates AI and synthetic biology to advance precision gene therapy.
16.06.25
Andrea Ballabio awarded Beth Levine Prize in Autophagy Research from UT Southwestern
Andrea Ballabio has been named the recipient of the 2025 Beth Levine, M.D. Prize in Autophagy Research from UT Southwestern Medical Center.
03.06.25
Long‑term dysfunction of edited blood stem cells and how to overcome it
SR‑Tiget researchers identify premature aging in CRISPR‑edited blood stem cells, but show that targeted interventions such as p53 inhibition and Anakinra can restore long‑term regenerative function.
28.05.25
A new window for in vivo blood‑stem‑cell gene therapy
A study from SR Tiget shows that blood stem cell gene therapy can be delivered directly in newborns during a short early life window.
24.04.25
Clinical Evidence Confirms Timing as Crucial in Gene Therapy for MLD
A NEJM study confirms that gene therapy, developed over 20 years at San Raffaele-Telethon, preserves motor and cognitive function in MLD patients if administered early.
31.03.25
From psoriasis to Lowe syndrome: clinical trial kicks off thanks to collaboration with Can-Fite
Research conducted at TIGEM has identified a molecule able to counteract renal dysfunction in this rare syndrome of genetic origin.
