Article

A Human Genetics Framework for De-risking Gene Editing Targets for Hematopoietic Cell and Gene Therapy

2025-12-17

Abstract excerpt

Developing novel therapeutics requires robust early-stage target de-risking to ensure safety and efficacy. We developed a scalable proteogenomic framework integrating population-scale human genetics and plasma proteomics to identify genes tolerant of inactivation (i.e., dispensable) within hematopoietic compartments, thereby enabling safer targeted immunotherapies. Using CD33 as a validated benchmark, we observed...

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Literature Corpus work: 788668e6-e5f7-57a5-af2e-e0af9814513a
DOI: 10.64898/2025.12.15.694499

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