Gene Therapy News -- ScienceDaily

03 March 2024

Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
  • Genetic signature may predict response to immunotherapy for non-small cell lung cancer
    A new study identified a set of 140 genes that may help predict enhanced disease-free survival in patients with non-small cell lung cancer (NSCLC) treated with a combination of immunotherapy and low-dose radiation. The results suggested that this 'gene signature' could be used to identify a subclass of lung tumors that is more likely to be eradicated by immunotherapies.
  • Gene-editing offers hope for people with hereditary disorder
    A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.
  • Researchers identify key characteristics associated with improved CAR T outcomes in large B cell lymphoma
    Axi-cel CAR T targets the CD19 molecule on large B-cell lymphoma cells. The ZUMA-7 trial demonstrated that axi-cel reduced the risk of disease progression, the need for new therapy, or death by 60% compared to standard therapy. Despite these positive outcomes in event-free survival and overall survival, some patients did not respond well to therapy or relapsed quickly after treatment. Researchers wanted to assess if there were specific tumor characteristics associated with improved outcomes that could better inform treatment selection.
  • A novel switch to turn genes on/off on cue, a promising step toward safer gene therapy
    Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect. Researchers now report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications.
  • Mice possess natural gene therapy system
    A previously mysterious small RNA molecule in mice is found to play a crucial role in gene expression, and may be the first identified member of a new class of regulatory RNAs.
  • Promising new gene therapy approach for genetic heart disease: clinical trials imminent
    Researchers have laid the foundation for the development of a gene therapy for the genetic heart disease arrhythmogenic cardiomyopathy (ACM). Their approach, based on replacement of the PKP2 gene, led to significant structural and functional improvements in laboratory models of the disease.
  • New target found for treatment of spinal muscular atrophy
    Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important limitations of gene therapy and other current therapies for SMA.