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Unlocking the Genome: Exploring CRISPR-Cas9 and Genotoxicity Concerns
Lavanya Venkatesan
Lavanya Venkatesan
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Unlocking the Genome: Exploring CRISPR-Cas9 and Genotoxicity Concerns

Exploring Gene Therapy and Genome Editing Technologies with a Focus on CRISPR-Cas9 Mechanism, Ethical Considerations, and Genotoxicity Studies

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Thursdays
 at
6:00
P.M.
 ET /
3:00
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

This project aims to explore key aspects of gene editing and gene therapy, focusing on current techniques, historical evolution, and the associated challenges. You will delve into the mechanisms and applications of gene editing technologies, understand how they have evolved from early experiments to sophisticated tools used in modern biotechnology and medicine. Utilizing bioinformatics tools, you will conduct an analysis to understand disease modalities, interpreting genomic data to identify and characterize structural variants, and assessing their potential impact on gene function and disease progression.  

The project will also investigate genomic instability and off-target effects that can arise from gene editing, emphasizing their implications for therapeutic safety and efficacy. Additionally, you will explore the role of bioinformatics as a powerful tool for deciphering disease modalities, leveraging computational approaches to analyze genomic data and understand disease mechanisms at a molecular level.

Through this comprehensive exploration, this Build Project seeks to equip you with a deep understanding of the complexities and potentials of gene editing technologies in healthcare and biotechnology. You will produce a report about the evolution and current state of gene editing techniques, including a historical overview of gene therapy. It will also cover the mechanisms of genomic instability and off-target effects resulting from gene editing, providing insights into their implications for therapeutic safety and efficacy.

Session timeline

  • Applications open
    August 1, 2024
  • Application deadline
    August 25, 2024
  • Project start date
    Week of July 8, 2024
    Week of
    September 9, 2024
  • Project end date
    Week of

What you will learn

  • Understand current gene editing techniques with an overview of the evolution and history of gene therapy
  • Understand genomic instability and off-target mediated effects that occur as a result of gene editing
  • Use Bioinformatics as an efficient tool to understand disease modalities  
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
Introduction and History of Gene Therapy
2
Current Gene Editing Techniques – Part 1
3
Current Gene Editing Techniques – Part 2
4
Off-Target Effects and Their Consequences
5
On-Target Effects and Efficacy
6
Genotoxicity and Safety Concerns
7
Ethical Considerations in Gene Editing
8
Gene Editing – An Overview

Prerequisites

  • A Basic understanding of Gene Editing Techniques: Knowledge of how gene editing tools like CRISPR/Cas9 and TALENs work at a basic level, including the concept of targeted DNA modifications.
  • Familiarity with the significance of gene therapy, its purpose, and basic history, including key milestones such as the first gene therapy trials.
  • A Basic understanding of the principles of genomic stability and instability, including basic concepts of DNA structure, function, and repair mechanisms.
  • Familiarity with the basic purpose and function of bioinformatics tools, such as sequence alignment and variant calling.
  • Basic ability to navigate and use tools like CRISPResso2 for analyzing sequencing data and UCSC Genome Browser for visualizing genomic data.

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About the expert
Lavanya Venkatesan
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Lavanya
's Linkedin
Throughout my life, I've realized that answering a question can sometimes take years of learning and understanding. From childhood queries about food and energy to finally comprehending the Krebs Cycle in high school, I've found immense satisfaction in unraveling scientific mysteries. These moments of insight fuel my passion for biotechnology, a field where my curiosity thrives and evolves. I got my Master's in Biological Sciences from Virginia Tech and I have been working for a Gene Editing start up as a researcher for the last three years. Very excited to be passing on some of my insights and experience to fellow curious researchers!