Researchers discovered that DNA repair determines how cancer cells die following radiotherapy, with specific pathways triggering cell death noticed by the immune system. Blocking these pathways can force cancer cells to die in a manner that alerts the immune system, leading to new potential treatments.
Scientists have successfully tested novel gene therapies in a whole human liver, opening up new avenues for treating life-threatening inherited diseases. The use of a normothermic liver perfusion system enables accurate testing of gene therapeutics directly in the clinical target organ.
The scientific community is urged to adopt more inclusive practices in preclinical and basic science research to promote personalized medicine. Researchers highlight the lack of diversity in commonly used human cell lines, which are predominantly of European descent, and emphasize the need for representative cell lines to better unders...
Researchers at Children's Medical Research Institute have discovered a reason behind the low success rate of gene therapy targeting liver diseases using Adeno-associated virus 2 (AAV2). The team found that AAV2 binds too tightly to its attachment receptor, heparan sulfate proteoglycans, which leads to the vector getting
Researchers at the Children's Medical Research Institute have identified syndapin as a crucial molecule in the transmission of messages between nerve cells. The partnership between syndapin and dynamin is vital for understanding neurological disorders such as epilepsy, conditions of memory loss and schizophrenia.