{"entity":{"id":"idea-bio1-somatic-crispr-gemms","kind":"idea","name":"Make bespoke mouse cancer models in weeks with in vivo gene editing","aka":[],"tldr":"Building a genetically engineered mouse for a specific cancer takes years. Editing genes directly in an adult mouse's organ can produce the same tumour in weeks.","summary":"Somatic genome editing by electroporation, viral delivery or lipid nanoparticles into a target organ produces autochthonous tumours with intact immune systems and native microenvironment, at a fraction of the time and cost of germline models. This has been demonstrated in lung, pancreas, liver and brain. Combinatorial guide libraries also allow genotype-response mapping in immunocompetent animals.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Lab models that fail to predict what happens in patients): Wong, Siah & Lo, Estimation of clinical trial success rates (Biostatistics 2019)","url":"https://doi.org/10.1093/biostatistics/kxx069"}],"tags":[],"related":[],"cancers":["nsclc","pancreatic","glioblastoma"],"sections":[],"technologies":["crispr-screens","pdx-models"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":["b-preclinical-models"],"keyPapers":["paper-wong-biostatistics"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"Somatic-editing models reproduce the histology, immune contexture and therapy response of matched germline models while reducing time to model from years to under three months.","rationale":"Immunocompetent, genotype-defined models are the scarcest resource in translational oncology; the editing tools now exist and the bottleneck is standardisation and sharing of protocols.","test":"Direct comparison of somatic and germline models for three well-characterised genotypes on histology, immune profiling and response to a standard agent.","maturity":"preclinical-evidence","actor":"research","cost":"small","horizonYears":3},"route":"/ideas/idea-bio1-somatic-crispr-gemms/","neighbours":{"cancer":[{"id":"glioblastoma","kind":"cancer","name":"Glioma & glioblastoma","route":"/cancers/glioblastoma/"},{"id":"nsclc","kind":"cancer","name":"Non-small-cell lung cancer","route":"/cancers/nsclc/"},{"id":"pancreatic","kind":"cancer","name":"Pancreatic ductal adenocarcinoma","route":"/cancers/pancreatic/"}],"technology":[{"id":"crispr-screens","kind":"technology","name":"CRISPR functional genomics","route":"/technologies/crispr-screens/"},{"id":"pdx-models","kind":"technology","name":"Patient-derived xenografts","route":"/technologies/pdx-models/"}],"bottleneck":[{"id":"b-preclinical-models","kind":"bottleneck","name":"Lab models that fail to predict what happens in patients","route":"/bottlenecks/b-preclinical-models/"}],"paper":[{"id":"paper-wong-biostatistics","kind":"paper","name":"Estimation of clinical trial success rates and related parameters","route":"/key-papers/paper-wong-biostatistics/"}]}}