Description
Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.
Gene Therapy covers all aspects of gene therapy as applied to human disease, including:
- preliminary studies which reveal novel genetic targets or strategies that improve a disease phenotype (Note: studies showing the mechanistic role of genetic elements alone, will be considered if significant therapeutic benefit or novelty is shown)
- the development of novel techniques for gene therapy research and improvements on established tools
- preclinical animal model systems and validation studies
- gene-based vaccine development and applications
- genetically engineered cellular models that can advance the validation of novel therapeutic targets, and drug discovery especially those using high-throughput applications and 3D modelling
- cell-based therapies including all aspects of stem cells and genetically modified cellular approaches (especially CRISPR and other gene editing strategies), including biobanking studies
- clinical trial reports which have significant impact for the field
- novel genetic biomarkers of disease susceptibility or disease progression with extensive scientific validation
- Industry sector pipeline profiles or commercial implications of gene therapies
- Regulatory landscapes and legislative issues pertaining to different global regions