Genetics and the Future of Medicine: Cystic Fibrosis and CRISPR Solutions

Generated from prompt:

Genetics & the Future of Medicine Project How can genetic engineering be used to understand, treat, or potentially cure inherited human disorders? In this project, you will research a genetic disorder or disease and explore how it is inherited, how it affects the body, and how modern science (genetic engineering) could be used to treat or cure it. You will create a final presentation that explains your findings and proposes a solution based on real scientific ideas. Part 1: Understanding the Disorder You must research and explain: The name of the disorder What causes it (gene, chromosome, or mutation) The type of inheritance: Dominant Recessive Sex-linked Chromosomal Who is affected (age, population, frequency) Clearly explain how the disorder is passed down Recommended to Include: Include a Punnett square OR pedigree chart Part 2: Molecular Biology Connection Explain what is happening inside the body at the molecular level: What type of mutation occurs? How does this mutation affect DNA and proteins? How does that lead to the symptoms? You must include: A clear explanation of: DNA → Protein → Trait (or disorder) Part 3: Symptoms & Impact Describe how the disorder affects a person: Physical symptoms Effects on body systems Daily life challenges Part 4: Current Treatments Research what is currently being done: Are there treatments available? Are there cures? What are the limitations of current treatments? Part 5: Genetic Engineering Solution Now you become the scientist. Design a possible treatment or cure using genetic engineering. You should answer: How would you fix or replace the faulty gene? What technology could be used? (ex: gene editing, gene therapy) What would your solution do inside the body? Your idea should be: Scientifically realistic (based on what we’ve learned) Clearly explained Part 6: Ethical Considerations Think beyond the science: Should this type of genetic engineering be used in humans? What are the risks? Who should have access to these treatments? You must include your opinion AND reasoning. Final Presentation You may choose ONE: Google Slides or Canva Poster / Infographic Short video Required Presentation Components Your project must include ALL of the following: ✔ Disorder overview ✔ Inheritance explanation (Punnett square or pedigree) ✔ Molecular explanation (DNA → protein → trait) ✔ Symptoms and impact ✔ Current treatments ✔ Genetic engineering solution ✔ Ethical discussion Rubric (60 Points Total) Category Description Points Disorder & Heredity Accurate explanation of disorder, cause, and inheritance pattern (includes Punnett square or pedigree) 10 pts Molecular Biology Clear explanation of mutation and how it affects DNA → protein → trait 5 pts Symptoms & Impact Thorough description of how the disorder affects the body and daily life 10 pts Current Treatments Accurate explanation of existing treatments and limitations 5 pts Genetic Engineering Solution Creative, realistic, and well-explained solution using genetic engineering 15 pts Ethical Considerations Thoughtful discussion with clear reasoning and support 5 pts Presentation Quality Organization, clarity, visuals, and effort 10 pts Total: 60 Points *You are REQUIRED to include a slide with all of the websites you used as resources. This is a research project so you should have multiple sources to support your presentation. write this slideshow at a 8th grade level and make it look human written

This 8th-grade presentation on Cystic Fibrosis covers the genetic disorder's overview, autosomal recessive inheritance via Punnett square, molecular biology link from CFTR gene mutations to thick mucus, symptoms impacting lungs and digestion, current treatments like modulators and their limits, a proposed CRISPR gene therapy workflow to repair the gene, ethical pros and cons, key statistics, and sources, concluding with hope for a cure.

May 12, 202618 slides
Slide 1 of 18

Slide 1 - Genetics & the Future of Medicine

Genetics & the Future of Medicine

Cystic Fibrosis Project - 8th Grade Presentation

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Photo by Sangharsh Lohakare on Unsplash

Slide 1 - Genetics & the Future of Medicine
Slide 2 of 18

Slide 2 - Presentation Agenda

  • Cystic Fibrosis Overview
  • Inheritance Pattern (Punnett Square)
  • Molecular Biology Connection
  • Symptoms & Impact
  • Current Treatments
  • Genetic Engineering Solution
  • Ethical Considerations
  • Sources

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Photo by Emmanuel Edward on Unsplash

Slide 2 - Presentation Agenda
Slide 4 of 18

Slide 4 - Cystic Fibrosis Overview

  • Cystic Fibrosis (CF) is a genetic disease that makes mucus thick and sticky in lungs, pancreas, liver, and intestines.
  • Caused by mutations in the CFTR gene on chromosome 7.
  • Autosomal recessive: Kids get it only if both parents pass on a faulty copy.
  • Affects 1 in 3,500 Caucasian newborns; less common in other groups.
  • Mostly kids and young adults - can shorten life if not treated.
Slide 4 - Cystic Fibrosis Overview
Slide 5 of 18

Slide 5 - Inheritance: Autosomal Recessive

  • Both parents are carriers (have one good and one faulty gene).
  • Each parent has 50% chance to pass faulty gene.
  • 1 in 4 kids will have CF (two faulty genes).
  • 2 in 4 will be carriers like parents.
  • 1 in 4 will be normal (no faulty genes).

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Photo by Sangharsh Lohakare on Unsplash

Source: Wikipedia: Cystic Fibrosis

Slide 5 - Inheritance: Autosomal Recessive
Slide 6 of 18

Slide 6 - Part 2

2

Molecular Biology Connection

How a tiny DNA change causes big problems

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Photo by Logan Voss on Unsplash

Slide 6 - Part 2
Slide 7 of 18

Slide 7 - DNA → Protein → Disorder

  • Normal DNA makes good CFTR protein (chloride channel).
  • Mutation (most common: Delta F508 deletion) makes faulty protein.
  • Faulty protein doesn't work right - can't move chloride ions.
  • Cells make thick mucus instead of thin slippery kind.
  • This clogs lungs and organs, causing infections and problems.

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Photo by Google DeepMind on Unsplash

Source: Wikipedia: CFTR

Slide 7 - DNA → Protein → Disorder
Slide 8 of 18

Slide 8 - Part 3

3

Symptoms & Impact

How CF affects the body and life

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Photo by volant on Unsplash

Slide 8 - Part 3
Slide 9 of 18

Slide 9 - Symptoms of Cystic Fibrosis

  • Lungs: Thick mucus causes cough, infections, trouble breathing. Can lead to lung damage.
  • Digestive: Can't digest food well - poor growth, greasy stools, malnutrition.
  • Salty sweat - easy to spot with sweat test.
  • Daily life: Lots of meds, breathing treatments, special diet, miss school sometimes.
  • Without care, life expectancy was short; now many live to 40+ with treatments.
Slide 9 - Symptoms of Cystic Fibrosis
Slide 10 of 18

Slide 10 - Part 4

4

Current Treatments

What's available now and its limits

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Photo by Haley Lawrence on Unsplash

Slide 10 - Part 4
Slide 11 of 18

Slide 11 - Treatments Today

  • No cure yet, but good symptom management.
  • Airway clearance (vests, physio) to loosen mucus.
  • Antibiotics for infections.
  • Enzyme pills to help digest food.
  • Modulator drugs like Trikafta help 90% of patients - make protein work better.
  • Limits: Expensive, daily routine, doesn't fix gene, lungs still damage over time.

Source: Cystic Fibrosis Foundation

Slide 11 - Treatments Today
Slide 12 of 18

Slide 12 - Part 5

5

Genetic Engineering Solution

My idea to fix it with science

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Photo by Sangharsh Lohakare on Unsplash

Slide 12 - Part 5
Slide 13 of 18

Slide 13 - My CRISPR Gene Therapy Plan

StepWhat It Does
1. Target CellsUse a safe virus to deliver CRISPR to lung cells.
2. Cut Bad GeneCRISPR cuts out the Delta F508 mutation in CFTR gene.
3. Repair DNAInsert correct DNA sequence to fix the gene.
4. Make Good ProteinCells now produce working CFTR protein.
5. Clear MucusThin mucus, fewer infections, healthier lungs.

Source: Nature: CRISPR for CF trials

Slide 13 - My CRISPR Gene Therapy Plan
Slide 14 of 18

Slide 14 - Part 6

6

Ethical Considerations

Should we do this? My opinion

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Photo by Memento Media on Unsplash

Slide 14 - Part 6
Slide 15 of 18

Slide 15 - Ethics: Pros, Cons, and My View

Risks & Concerns Off-target edits could cause new diseases. Expensive - only rich people get it? Germline editing changes kids forever. Slippery slope to 'designer babies'.

Benefits & My Opinion Yes, we should use it for sick people! Saves lives, ends suffering for CF families. Somatic editing (not germline) is safer. Everyone deserves a shot at cure if safe.

Slide 15 - Ethics: Pros, Cons, and My View
Slide 16 of 18

Slide 16 - Key CF Stats

  • 1/3500: Caucasian Births Affected
  • 40+: Median Life Expectancy
  • 90%: Helped by Trikafta
  • 30K: People with CF in US

Source: CFF.org

Slide 16 - Key CF Stats
Slide 17 of 18

Slide 17 - Sources

  • https://en.wikipedia.org/wiki/Cystic_fibrosis
  • https://www.cff.org/What-is-CF/What-is-CF
  • https://medlineplus.gov/genetics/condition/cystic-fibrosis/
  • https://www.nature.com/articles/s41591-023-02296-6 (CRISPR trials)
  • https://www.nhlbi.nih.gov/health/cystic-fibrosis
Slide 17 - Sources
Slide 18 of 18

Slide 18 - Wrapping Up

CF is tough, but genetic engineering offers real hope for a cure! Science + ethics = better future.

Thanks for listening! Any questions?

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Photo by Tobias Tullius on Unsplash

Slide 18 - Wrapping Up

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