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A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant traits in a mouse model, according to a new study published in Gene Therapy.

Preclinical trial is testing the feasibility of humanoid robots in surgery. O recorte ajuda a contextualizar a pauta dentro de Tech.

A new study led by researchers at The University of Texas MD Anderson Cancer Center has identified a way to tailor drug combinations based on specific tumor biology to improve outcomes for treatment-resistant advanced melanoma.

Researchers at the University of Pittsburgh, UPMC Hillman Cancer Center and the National Cancer Institute report that combining CAR T cell immunotherapy with a targeted radiopharmaceutical significantly improved tumor regression in preclinical models of neuroblastoma, a rare and

Scientists at UT Southwestern Medical Center have discovered that gut bacteria help regulate the development of the body's immune system by directing the movement of vitamin A through a previously unrecognized cellular network. The preclinical findings, published in Cell Host & M

Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), announced the publication of preclinical data supporting the potential of its liver-directed immune-shielded lentiviral gene therapy approach to treat methylmalonic ac

Hereditary primary hemochromatosis is caused by a single faulty building block in a gene. This leads to iron overload, which can have serious consequences for organs and joints. In preclinical studies, researchers have already successfully treated this genetic defect using a targ

An update on the first clinical trial evaluating a pluripotent stem cell-derived neural stem cell therapy for Huntington's disease was presented at the ISSCR 2026 Annual Meeting. The Phase Ib/IIa REGEN4HD study represents a significant translational milestone following years of p

Inhibiting menin, a protein that supports leukemia growth and is already targeted to treat some forms of leukemia, also holds promise for treating myeloproliferative neoplasms. A new study from scientists at St. Jude Children's Research Hospital showed that inhibiting menin signi

New research from VCU Massey Comprehensive Cancer Center provides evidence for a combination treatment strategy that uses cells' waste-removal functions to target multiple myeloma. The findings, published recently in Cell Death & Disease, show how an experimental targeted protein

An international research group led by researchers from VUB-UZ Brussels and KU Leuven has demonstrated that a combined approach targeting both the immune system and insulin-producing beta cells can slow the progression of type 1 diabetes more effectively than individual treatment

Wearable devices have transformed how people track exercise, sleep, heart rate and other vital signs. Researchers at King Abdullah University of Science and Technology (KAUST) are now exploring whether similar technologies could one day help monitor medicines inside the body.