Scientist​/Associate Scientist – Preclinical Design & Development; PDD) Core

Join our Team! The Preclinical Design and Development (PDD) Core within the Pediatric Translational Neuroscience Initiative e Children's Research Hospital

The Preclinical Design and Development (PDD) Core within the Pediatric Translational Neuroscience Initiative e Children's Research Hospital is seeking a rigorous, execution-focused Scientist with a strong background in patient-derived cell model development, high-throughput screening, and RNA biology. The candidate will execute cellular RNA-therapeutic evaluation workflows across a portfolio of cases with diverse genomic underpinnings. This is an exciting opportunity for a scientist seeking a team-based, project-driven role at a leading research institution.

The PDD core advances mutation-specific antisense therapies for children with ultra-rare and even one-of-a-kind (n‑of‑1) genetic conditions. Working at the cutting edge of precision genomics and translational medicine, the goal is to move rapidly from a child’s genetic diagnosis to a potential therapeutic candidate, with rigor, urgency, and purpose.

The ideal candidate is a skilled molecular biologist with expertise in plate-based cellular screening workflows and working knowledge of splicing/RNA biology. We are looking for a collaborative, methodical scientist who is self-directed and at ease with the iterative, fast‑paced nature of translational work.

• Experience with plate-based molecular screening workflows and quantitative nucleic acid assays (RT‑qPCR, ddPCR); familiarity with isoform-discriminating or allele‑specific probe design is preferred.
• Working knowledge of RNA biology, particularly pre‑mRNA splicing, and comfort interpreting molecular readouts in a therapeutic context.
• Proficiency in mammalian cell culture, including complex or patient‑derived cellular models.
• Ability to manage and execute multi‑step experimental workflows across parallel projects with consistency and rigor.
• Comfort adapting established protocols to new cellular contexts and working through scientific uncertainty iteratively.
• Team‑first orientation and comfort with flexible, pipeline‑driven translational work.

• Design and execute cell‑based therapeutic evaluation workflows and validation pipelines with high rigor across concurrent patient cases.
• Derive, characterize, and bank patient‑specific cellular models to enable screening and functional validation.
• Design and validate quantitative molecular assays (RT‑qPCR, ddPCR) and perform biochemical and imaging‑based readouts to evaluate therapeutic efficacy.
• Manage parallel experimental timelines and maintain proactive communication with collaborating teams to support project objectives.
• Maintain rigorous documentation, data integrity, and quality control standards consistent with a Core laboratory environment.
• Support monitoring and stocking of supplies; calibration, maintenance, or troubleshooting of equipment.
• Contribute positively to a culture of shared responsibility, mutual support, and collective scientific progress.
• Perform other duties as assigned in support of Core and departmental objectives.

Bachelor’s degree in relevant scientific area, such as genomics, molecular biology, cell biology, is required. Prior experience with Janus liquid handlers is a plus.

• Eight (8) years of relevant work experience is required.
• Six (6) years of relevant work experience is required with a Master’s degree.
• Three (3) years of relevant work experience is required with a PhD.

• 7+ years of research experience is required.
• 5+ years of research experience is required with a Master’s degree.
• 2+ years of research…