Cell Biology Lead - CRISPR Cures Technology Core

About Berkeley

At the University of California, Berkeley, we are dedicated to fostering a community where everyone feels welcome and can thrive. Our culture of openness, freedom and belonging make it a special place for students, faculty and staff.

The Innovative Genomics Institute is a joint effort between the Bay Area's leading scientific research institutions, UC Berkeley and UC San Francisco, with affiliates at UC Davis, Lawrence Berkeley National Laboratory, Lawrence Livermore National Laboratory, Gladstone Institutes, and other institutions. The IGI's diverse group of leading scientists have powerful interdisciplinary expertise. They conduct world-class research, driven by the real possibility of using genome engineering to treat human diseases, end hunger, and respond to climate change.

In addition to our scientific efforts, the IGI is committed to advancing public understanding of genome engineering, providing resources for the broader community, and guiding the ethical use of these technologies.

Reporting to the Director of the Biohub-IGI Center for Pediatric CRISPR Cures, the SRA3 performs experimental work aimed at advancing to first‑in‑human clinical trials CRISPR‑based approaches to treat inborn errors of immunity. Working in close partnership with, and supporting, the effort of physician‑scientists leading the nonclinical efforts in the Beacon, the SRA3 is responsible for the design, prosecution, and analysis of experiments assessing the potency, specificity, and biological activity of candidate therapeutic gene‑editor compositions.

The Innovative Genomics Institute has established a Center for Pediatric CRISPR Cures. Its goal is to develop and advance to the clinic gene‑editing‑based therapies for two severe inborn errors of the immune system. The SRA3 represents a linchpin position within the nonclinical (IND‑enabling) component of the effort in both leading on, and executing, on a range of experiments to establish the efficacy and safety of therapeutic candidate gene editors, and, when scientifically relevant, the design, development, and optimization of assays to assess efficacy/safety.

• Perform experiments in primary and transformed human cells to assess gene editor efficacy. Experimentally identify on a cell‑type‑by‑cell‑type basis optimal cell husbandry conditions for gene editing experiments. Optimize tissue culture conditions for maximal viability post‑gene editor transfer. Develop and perform cell phenotyping assays to evaluate physiological consequences of gene editing. Isolate cellular material (protein, RNA, DNA) for downstream analytics optimizing for yield and sample integrity.
• Perform experiments in primary and transformed human cells to assess gene editor safety. Optimize cell husbandry conditions for dose‑response‑curve experiments in primary and transformed human cells. Develop and deploy cell phenotyping assays to assess on‑target engagement following gene editor delivery. Isolate nucleic acids (DNA and RNA) from gene‑edited cell preparations. Prepare nucleic acid libraries for off‑target assessment. Optimize and deploy computational procedures to determine genome/transcriptome‑wide consequences of gene editing.
• Assess gene editing outcomes using established cell‑ and nucleic‑acid‑based analytical pipelines. Following introduction of gene editors into primary and transformed human cells, use cell sorting and, where appropriate, microscopy‑based procedures to characterize the cells at the phenotypic level. Extract nucleic acids from gene‑edited cells and assess gene editing efficiency using NGS, ddPCR, and RT‑qPCR methods.
• Design, develop, and prosecute novel analytical procedures to assess gene editing outcomes. Identify project‑critical analytical endpoints required to address Agency feedback. Working closely with project nonclinical leads and analytical core lead, identify suite of assays to be developed to measure these endpoints. Iterate on a design‑build‑test process to reduce the assays to operational practice.
• Supervise junior personnel in prosecution of efficacy/safety/analytical studies. On an as‑need…