Risk assessment in gene therapy and somatic genome-editing: An expert interview study

Innovation in gene therapy and genome editing raises high expectations for therapeutic breakthroughs. With the increasing maturity of field, products using “gene transfer” technology, such as viral vectors, designer nucleases, incl. CRISPR/Cas, most recent, are frequently tested clinical trials. Before trials launched, anticipated risks benefits transfer technologies must be evaluated to ascertain an ethical balance benefits. We conducted semi-structured interviews with experts (n=15) therapy/genome editing. applied thematic text analysis identify qualitative spectrum strengths, weaknesses, opportunities, threats (SWOT) a risk assessment approach research based on comprehensive set nine mechanistic categories adverse reactions combined estimates probability according World Health Organization (WHO) reaction terminology. Our study revealed clear demand structured The indicate that presented may helpful structure this prior initiating new study. broad practice-oriented SWOT, described detail manuscript. here SWOT inform refinement implementation standardized approaches discussion among researchers, regulators, funders. To overcome potential weaknesses application risk-based approach, need case-sensitive complemented by information validity relevant animal models, long-term risks, about patient characteristics.