Gene Editing & Molecular Analysis Services

The contract involves the execution of CRISPR/Cas9 or equivalent gene editing techniques to correct mutations in splicing factors within human cell models, with the goal of restoring normal RNA splicing function. This work requires precise genomic targeting, efficient delivery of editing components, and rigorous optimization to achieve high editing rates while minimizing off-target effects. All editing procedures must be carried out using validated protocols compliant with current best practices in gene editing and cell culture integrity. Following gene correction, comprehensive functional assays and next-generation sequencing analyses are required to validate the molecular and phenotypic outcomes. Validation must include assessment of splicing correction at the RNA level, confirmation of on-target edits with deep sequencing, and evaluation of cellular function to demonstrate restoration of physiological relevance. The contractor is expected to deliver detailed analytical reports, raw sequencing data, and a clear demonstration of successful correction and functional recovery in the modeled system, with all work to be completed by the response deadline of July 9, 2026, under a subcontract awarded by the National Institutes of Health under the Department of Health and Human Services.