GENOME

EDITING
TECNOLOGIES

SHIVANSH MANOJ
2021BB10334
INTRODUCTION
In today's rapidly advancing scientific landscape,
genome editing technologies stand at the
forefront of groundbreaking innovations.

Genome editing refers to the precise

modification of an organism's DNA, allowing
scientists to add, delete, or replace specific DNA
sequences. This revolutionary field holds
immense promise, not only in the realm of
scientific research but also in addressing critical
challenges in healthcare, agriculture, and
beyond. So, let's dive in and explore the
fascinating world of genome engineering!
 WHAT IS GENOME EDITING?

Genome editing, at its core, is the precise

alteration of an organism's DNA, the hereditary
material that carries the instructions for building
and maintaining an organism. This technology
enables scientists to make controlled and
specific changes in the genetic information of
an organism, such as adding, deleting, or
replacing DNA sequences. Genome editing tools
act as molecular scissors, allowing us to edit the
genetic blueprint of life with unprecedented
precision.
 HISTORICAL CONTEXT
ZFNS
(ZINC FINGER NUCLEASES)
Developed in the early 2000s.
Use engineered proteins (zinc
finger proteins) to target
specific DNA sequences.
Pioneered the concept of
programmable DNA cleavage.
TALENS
(TRANSCRIPTION ACTIVATOR-LIKE
EFFECTOR NUCLEASES)
Emerged around 2010.
Use TALE proteins to guide
nuclease enzymes to precise
DNA locations.
Enhanced targeting
capabilities.
CRISPR-CAS9:
First described in the mid-2010s.
Utilizes RNA-guided Cas9
proteins to target and modify
DNA.
Simplicity, precision, and
versatility made CRISPR-Cas9 a
revolutionary leap in the field.
THE CRISPR-CAS9 SYSTEM
The CRISPR-Cas9 system is a game-changing genome editing technology
that has gained widespread attention and acclaim in the scientific
community. In this slide, we will explore how CRISPR-Cas9 works and its key
applications in fields such as gene therapy and agriculture.

CRISPR-Cas9 is generally considered superior to ZFNs (Zinc Finger

Nucleases) and TALENs (Transcription Activator-Like Effector Nucleases)
due to its simplicity, precision, and versatility. Unlike the complex and time-
consuming design of ZFNs and TALENs, CRISPR-Cas9 utilizes a
straightforward RNA guide to target DNA sequences, making it easier to
engineer. Its precision and ability to target multiple genes simultaneously
also set it apart, while ZFNs and TALENs often target single genes.
Additionally, CRISPR's wide adoption and extensive research have led to a
more robust and accessible toolbox for genome editing, offering greater
potential for scientific and medical applications.
HOW IT WORKS ?
1. CRISPR SEQUENCES

2. CAS9 PROTEIN

3. ADAPTATION

4. TRANSCRIPTION AND PROCESSING

5. RECOGNITION AND CLEAVAGE

6. DNA CLEAVAGE

7. DNA REPAIR MECHANISMS

8. DESIRED GENETIC CHANGES

9. FUNCTIONAL OUTCOMES
 APPLICATIONS IN HUMAN HEALTH

Gene Therapy and Curing Genetic Diseases

CRISPR-Cas9 enables gene therapy.
It targets and repairs malfunctioning genes, offering potential cures.
Promising clinical trials for diseases like sickle cell anemia, cystic fibrosis, and muscular
dystrophy.
Enhanced quality of life for affected individuals.

Ethical Considerations
CRISPR's power raises ethical concerns.
Debate over misuse, designer babies, and germline editing.
Vital ethical guidelines for responsible use and individual rights.

Recent Breakthroughs and Case Studies

Notable successes in gene editing.
E.g., treating hereditary blindness (Leber congenital amaurosis) with CRISPR.
Demonstrates rapid progress in clinical applications and life-changing potential for
genetic diseases.
 APPLICATIONS IN AGRICULTURE

Enhanced Crops with Improved Nutritional Value

CRISPR precision enhances crop nutrition, fortifying staple foods with vital nutrients.
Biofortification projects address global malnutrition, targeting iron, zinc, and vitamin
content.
Nutrient-rich crops contribute to better health outcomes and food security.

Resistance to Pests and Diseases

CRISPR technology empowers crops with innate resistance to pests and diseases.
Reduced reliance on chemical pesticides, reducing environmental harm.
Enhanced crop protection ensures more reliable harvests and reduced food waste.
Enhanced disease resistance promotes greater crop longevity, reducing the need for
replanting and conserving land resources.

Sustainable Agriculture Practices

CRISPR-enabled resource-efficient crops require less water and fertilizers.
Precision breeding helps create drought-resistant varieties, preserving water resources.
Sustainable practices driven by CRISPR offer long-term solutions for global food
production and environmental conservation.
 ENVIRONMENTAL IMPLICATIONS

Gene Editing for Conservation

Gene editing aids in conservation efforts.
Preserves endangered species and restores ecosystems.
Enhances resilience of wildlife and plants.

Potential Risks and Benefits

Benefits include biodiversity restoration and carbon capture.
Risks involve ecological disruptions and unknown consequences.
Balancing benefits and risks is crucial.

Regulatory and Ethical Concerns

Gene editing in the environment demands robust regulations.
Oversight, containment, and misuse prevention are critical.
Ethical issues revolve around modified organisms and ecosystem
alterations.
 GENOME EDITING IN BIOTECHNOLOGY

Customized Microbes for Biofuels

Gene editing aids in conservation efforts.
Preserves endangered species and restores ecosystems.
Enhances resilience of wildlife and plants.

Creating Bio-Based Materials

Benefits include biodiversity restoration and carbon capture.
Risks involve ecological disruptions and unknown consequences.
Balancing benefits and risks is crucial.

Advancements in Pharmaceuticals
Gene editing in the environment demands robust regulations.
Oversight, containment, and misuse prevention are critical.
Ethical issues revolve around modified organisms and ecosystem
alterations.
 CHALLENGES AND ETHICAL CONCERNS

Off-Target Effects
Unintended gene alterations from genome editing.
Potential for unexpected consequences.
Need for improved precision and safety measures.

Germline Editing
Heritable DNA changes, impacting future generations.
Raises profound ethical and safety concerns.
Requires strict international regulations.

Informed Consent and Responsible Uses

Fundamental requirement in genome editing.
Comprehensive understanding of risks and benefits.
Transparent guidelines, oversight, and public engagement for responsible
use.
 CHALLENGES AND ETHICAL CONCERNS

Off-Target Effects
Unintended gene alterations from genome editing.
Potential for unexpected consequences.
Need for improved precision and safety measures.

Germline Editing
Heritable DNA changes, impacting future generations.
Raises profound ethical and safety concerns.
Requires strict international regulations.

Informed Consent and Responsible Uses

Fundamental requirement in genome editing.
Comprehensive understanding of risks and benefits.
Transparent guidelines, oversight, and public engagement for responsible
use.
 RISK VS REWARD

Balancing Benefits and Risks

Evaluate genome editing's benefits and ethical risks.
Strive for a balance that maximizes benefits and minimizes harm.

Case Studies
Examine real-world cases to show the complexity of decision-making.
Gene therapy, agriculture, and environmental examples.
Highlight the outcomes of responsible genome editing.

Public Perception
Public views influence genome editing's acceptance.
Address concerns, maintain transparency, and build trust.
Engage with the public and stakeholders to align with ethical
standards.
 CONCLUSION
1. Genome Editing's Revolution: Genome editing technologies, such as CRISPR-Cas9, have revolutionized the
fields of science, medicine, agriculture, and biotechnology. Their precision and versatility offer
unprecedented opportunities.
2. Applications with Impact: Genome editing is instrumental in gene therapy, disease treatment, sustainable
agriculture, and bio-based materials. It addresses critical global challenges.
3. Ethical Considerations: The power of genome editing comes with significant ethical concerns, including
germline editing and the responsible use of technology.
4. Environmental Implications: Genome editing has the potential to enhance conservation efforts, reduce
environmental impact, and tackle climate change.
5. Challenges and Risks: Unintended off-target effects, germline editing, and ethical concerns pose challenges
that demand careful navigation and strict regulations.
6. Balancing Act: Weighing benefits against risks is a complex task. Case studies reveal the intricacies of
decision-making in gene editing applications.
7. Public Engagement: Public perception and engagement play a significant role in shaping the acceptance
and ethical use of genome editing technologies.
8. The Road Ahead: As we move forward, responsible, transparent, and ethical genome editing
practices are pivotal in harnessing the technology's potential for the betterment of humanity and
the environment.
THANK YOU