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Identify Gene Targets and Develop Gene Editing Strategies for Cellular Therapies
Marie Goulard
Marie Goulard
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Identify Gene Targets and Develop Gene Editing Strategies for Cellular Therapies

Use popular industry tools to analyze an RNAseq gene dataset and propose useable Crispr guides and primers for a gene knockout.

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Mondays
 at
5:00
P.M.
 ET /
2:00
P.M.
PT
8 weeks, 2-3 hours per week
Intermediate
No experience required
No experience required
Some experience required
Degree and experience required

Description

Allogenic hematopoietic stem cell transplant (HSCT) is the only cure for patients with blood, bone marrow and immune disorders. Understanding the underlying molecular biology behind HSC function, expansion, and differentiation is critical to improve cellular therapies for multiple diseases. In this Build Project, you will wear the hat of a Molecular Research Associate to perform RNA sequencing analysis and then identify relevant pathways and genes of interest in HSC functions. All this will happen in an environment that simulates the operations of a real Research team.


By the end of the 8-week project, students will develop deliverables for this project are focused on building artifacts that you can use to showcase your work in a job application. By the end of the micro-internship, you'll review an RNAseq gene pathway from human hematopoietic stem cells, perform a comparative analysis between multiple conditions, and develop gene knockout and genotyping designs. In the final project session, you'll present your results to the Project Leader in a format that mimics a job interview (so you’re ready to discuss your work in real-life interviews).

Session timeline

  • Applications open
    June 24, 2024
  • Application deadline
    July 21, 2024
  • Project start date
    Week of July 8, 2024
    Week of
    August 5, 2024
  • Project end date
    Week of

What you will learn

  • Learn how to use the online platform basepair for next generation sequencing data analysis with publicly available RNA sequencing (RNAseq) data
  • Become familiar with DAVID and Enrichr, two popular industry tools, to identify gene ontology, pathways, and genes of interest
  • Learn how to validate the gene relevance and will create strategies to perform a genetic knockout of the selected gene(s) in stem cells to HSC biology using the online tools Ensembl and NCBI. 

Project workshops

1
Introduction
2
Hematopoietic Stem Cells in Cellular Therapies
3
New Models for Cellular Therapies
4
Gene Stability and RNAseq.
5
Open-source Databases and Tools for Gene Analysis
6
Crispr Cas9 Technology
7
Polymerase Chain Reaction (PCR)
8
Presentations

Prerequisites

  • Basic understanding of cell biology (notions of different cell types, concept of cell differentiation) 
  • Basic understanding of molecular biology (concept of RNA and DNA, PCR)
  • Intellectual curiosity about stem cells and blood formation
  • Attention to details  

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About the expert

Marie Goulard

Molecular Biology Fellow
Open Avenues Foundation

Marie has 10+ years experience with Stem Cells and Cell therapies including academia and biotech company. She is currently Director of Research and Development at Garuda Therapeutics, a biotech company in Cambridge, MA. Marie has worked across many therapeutic areas including Oncology, Hematology, and Cell Therapies. She holds a PhD in Oncology and Hematology for the University of Paris Diderot, France.

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