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Precision Guide Quest: Designing and Optimizing CRISPR/Cas9 Gene Editing Strategy
Harshi Gangrade
Harshi Gangrade
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Precision Guide Quest: Designing and Optimizing CRISPR/Cas9 Gene Editing Strategy

In this Project, students will focus on designing and optimizing a CRISPR/Cas9 gene editing strategies for a specific target gene leading to a comprehensive report on a plan for successful gene modification.

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Thursdays
 at
2:00
P.M.
 ET /
11:00
A.M.
PT
8 weeks, 2-3 hours per week
Intermediate
No experience required
No experience required
Some experience required
Degree and experience required

Description

Embark on Precision Guide Quest!  CRISPR/Cas9 gene editing revolutionized the biotech industry with its precise and versatile approach to modifying DNA. This technology utilizes a guide RNA to direct the Cas9 enzyme to specific genomic locations, where it induces double-strand breaks that can be repaired to introduce desired genetic changes. CRISPR/Cas9 has become indispensable in various industries, particularly in biomedicine, agriculture, and biotechnology, due to its ability to precisely edit genes, model diseases, develop novel therapeutics, and improve crop traits. Its efficiency, affordability, and scalability have propelled it to the forefront of genetic engineering, promising breakthroughs in treating genetic disorders, enhancing crop resilience, and advancing personalized medicine.

In this Build Project, you will create a meticulous gene editing strategy using CRISPR/Cas9 for a specific target gene crucial in disease research. You will design guide RNAs to precisely direct the Cas9 enzyme to the desired genomic location, enabling them to induce precise genetic modifications. Additionally, you will explore and select optimal delivery methods to introduce the CRISPR/Cas9 components into target cells or organisms, ensuring efficient editing while minimizing off-target effects with the insights in in vivo studies. Through critical thinking and problem-solving skills, you will develop a comprehensive understanding of CRISPR/Cas9 editing strategies and gain valuable skills applicable to the biotech industry. You will be creating a pitch deck at the end of the project which will showcase your skillset with a flow of understanding and skills learnt in this project which are the gateway to the coveted roles in the biotech industry and will act as a catalyst for a rewarding career at the forefront of genetic innovation!

Session timeline

  • Applications open
    May 27, 2024
  • Application deadline
    June 23, 2024
  • Project start date
    Week of July 8, 2024
    Week of
    July 8, 2024
  • Project end date
    Week of

What you will learn

  • Understand the target gene and its protein expression underlying the disease using NCBI gene database and other Bioinformatic Tools.
  • Successfully design gRNA using CRISPOR, Chop-Chop and Snapgene by understanding the CFD score and specificity.
  • In silico analysis of off-target sites using CRISPR-OT.
  • Conceptualize Delivery methods and Experimental condition i.e. Transfection/Transduction  
  • Understand validation strategies like PCR assays, Functional assays (ELISA/Western Blotting).
  • Understand the Future in vivo studies to validate gene editing.
Build Projects are 8-week experiences that operate on a rolling basis. Selected participants engage in weekly live workshops with a Build Fellow and 2-15 other students.

Project workshops

1
Introductions
2
gRNA designing
3
Delivery methods: Transfection and transduction
4
Designing of Experiment (DOE)
5
PCR assay and analysis, NGS sequencing
6
Functional assays
7
In vivo insights, Ethical considerations
8
Project presentations, Reflections and questions about my role/company

Prerequisites

  • Familiarity with basic concepts of Molecular biology and Cell biology.
  • Understanding of DNA structure, Genetic expression, Protein Expression, translation, and transcription processes.  
  • Knowledge of Basic cell culture techniques like Handling and maintenance of different cell types.
  • Ability to use bioinformatics tools and interpret the results and draw conclusions for the experimental outcomes.  
  • Capacity to identify challenges and devise strategies for optimizing the processes.

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About the expert
Harshi Gangrade
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Harshi
's Linkedin

I am Harshi Gangrade, a senior research associate at CRISPR Therapeutics. My focus is on manipulating the expression of the Angiotensinogen gene to manage refractory hypertension. Originally from India, I completed my Bachelor’s in Biotechnology at D.Y. Patil University before pursuing my Masters in Biotechnology at Johns Hopkins University in the United States, graduating in 2020. Following my studies, I conducted research under Dr. Emmanouil Tampakakis in a cardiology lab at Johns Hopkins Medical Institute, where I contributed to several projects and authored multiple research papers. After two years, I transitioned to industrial research to gain insights into the commercial aspects and the translation of biotechnology from laboratory experiments to practical applications. Joining CRISPR Therapeutics in 2022 has provided me with valuable opportunities in this regard, and I have found great satisfaction in my work here ever since.

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