Research Intern

This role is designed to be 100% onsite, giving you the opportunity to collaborate closely with your team and immerse yourself in the energy of our workplace. Please note that hybrid or remote work options are NOT available for this position at this time.

Educational Value:

This internship provides valuable exposure to translational neuroscience, rare disease modeling, and stem-cell-based discovery. Interns will leave with a solid foundation in neurogenetics, molecular biology, and stem cell research, preparing them for future graduate, medical, or research career paths.

The Board of Governors Regenerative Medicine Institute (RMI) at Cedars-Sinai Medical Center develops and applies cutting-edge stem cell technologies to deepen our understanding of human diseases across multiple organ systems. RMI’s research integrates stem-cell-based modeling platforms to replicate molecular events that drive disease onset, providing powerful tools for drug discovery and the identification of novel therapeutic targets.

Overview of Mentor:

The intern will be mentored by a pediatric neurologist and researcher with over 20 years of laboratory and clinical experience in neurogenetic disorders. The mentor’s research focuses on rare and ultra-rare childhood disorders, particularly lysosomal diseases and gene regulation abnormalities, combining clinical insight with translational molecular science.

Overview of the Laboratory:

Our lab investigates rare neurogenetic disorders caused by genetic aberrations that disrupt the function of the central or peripheral nervous system. Because these genes are vital for neurodevelopment and human physiology, their malfunction often manifests early in life, resulting in severe and sometimes life-threatening conditions. Studying these rare disorders not only benefits affected patients but also provides key insights into broader biological mechanisms. Such discoveries have historically led to breakthroughs in understanding and treating more common diseases.

Role Summary and Objectives:

This internship offers an educational experience in the study of rare pediatric neurogenetic disorders, combining data collection, analysis, and laboratory-based research using induced pluripotent stem cell (iPSC) models.

Goals:

• Learn the fundamentals of neurogenetic disorder research.
• Gain hands-on experience with molecular biology techniques and iPSC modeling.
• Participate in data collection, genetic manipulation, and analysis for rare neurodevelopmental disorders.

Objectives:

The intern will learn to collect and analyze phenotypic data, culture and manipulate iPSC models, and apply molecular biology methods such as CRISPR-based editing. Projects may include studies of GATAD2B-associated neurodevelopmental disorder (GAND) and other NuRD-related conditions.

Project Scope & Training Timeline:

Months 1–2:

• Learn about neurogenetic disorders and neurological phenotypes.
• Organize phenotypic and genetic data using spreadsheets.
• Observe and document patient interviews under the supervision of the principal investigator (PI).
• Learn to contact families, schedule interviews, and collect neurodiagnostic data.

Months 2–3:

• Begin analyzing patient data in collaboration with the PI.
• Receive instruction in writing the Introduction and Methods sections of a research paper.

Months 1–4 (Wet Lab Training):

• Learn molecular biology and tissue culture techniques, including iPSC maintenance and DNA plasmid cloning.
• Participate in designing and executing experiments using control and disease-specific iPSC and cerebral organoid models.
• Perform immunocytochemistry (ICC) and staining experiments to study neuronal differentiation and gene expression.
• Implement CRISPR protocols to create isogenic or genetically altered cell lines.
• Contribute to single-cell transcriptomic analyses and differentiation marker studies.