Podcast
Questions and Answers
Which of the following best describes the primary focus of genetics as a field of study?
Which of the following best describes the primary focus of genetics as a field of study?
- The investigation of chemical processes within living organisms.
- The study of the structure and function of cells.
- The branch of biology that studies genes, genetic variation, and heredity in living organisms. (correct)
- The study of past civilizations and their DNA.
A gene is best described as a segment of RNA that acts as a blueprint for building and regulating proteins.
A gene is best described as a segment of RNA that acts as a blueprint for building and regulating proteins.
False (B)
Define heredity in the context of genetics.
Define heredity in the context of genetics.
Heredity is the process by which traits and characteristics are passed from one generation to the next.
Gregor Mendel's experiments with ________ plants revealed how traits are passed from parents to offspring, establishing the basis for Mendelian inheritance.
Gregor Mendel's experiments with ________ plants revealed how traits are passed from parents to offspring, establishing the basis for Mendelian inheritance.
Which of the following is NOT a cause of genetic variation?
Which of the following is NOT a cause of genetic variation?
Gene therapy primarily focuses on managing the symptoms of genetic conditions rather than addressing their underlying genetic causes.
Gene therapy primarily focuses on managing the symptoms of genetic conditions rather than addressing their underlying genetic causes.
Briefly describe one potential application of gene therapy.
Briefly describe one potential application of gene therapy.
In gene therapy, ___________ a mutated gene involves inactivating or 'silencing' it to prevent it from causing problems.
In gene therapy, ___________ a mutated gene involves inactivating or 'silencing' it to prevent it from causing problems.
What is the most significant advantage of gene therapy in treating genetic disorders compared to traditional treatments?
What is the most significant advantage of gene therapy in treating genetic disorders compared to traditional treatments?
Gene therapy reduces dependency on lifelong medications or surgeries.
Gene therapy reduces dependency on lifelong medications or surgeries.
Why is gene therapy considered a step towards personalized medicine?
Why is gene therapy considered a step towards personalized medicine?
In _______ (year), Luxturna became the first gene therapy approved by the FDA for treating inherited retinal disease.
In _______ (year), Luxturna became the first gene therapy approved by the FDA for treating inherited retinal disease.
Which of the following statements best describes 'ex vivo' gene therapy?
Which of the following statements best describes 'ex vivo' gene therapy?
In 'in vivo' gene therapy, cells are removed, modified in the laboratory, and then reintroduced into the patient.
In 'in vivo' gene therapy, cells are removed, modified in the laboratory, and then reintroduced into the patient.
Describe the key difference between 'in vivo' and 'ex vivo' gene therapy approaches.
Describe the key difference between 'in vivo' and 'ex vivo' gene therapy approaches.
In 'in vivo' gene therapy, the ___________ gene is directly delivered into the patient’s body, targeting specific cells or tissues.
In 'in vivo' gene therapy, the ___________ gene is directly delivered into the patient’s body, targeting specific cells or tissues.
Which of the following is commonly used as a vector in gene therapy to deliver therapeutic genes to target cells?
Which of the following is commonly used as a vector in gene therapy to deliver therapeutic genes to target cells?
CRISPR-Cas9 technology is a method used for genome editing that allows for highly precise treatment.
CRISPR-Cas9 technology is a method used for genome editing that allows for highly precise treatment.
Identify a significant ethical issue associated with gene therapy.
Identify a significant ethical issue associated with gene therapy.
One of the challenges of gene therapy arises from ______________ of vectors in gene therapy, where the immune system might react adversely to the introduced vectors.
One of the challenges of gene therapy arises from ______________ of vectors in gene therapy, where the immune system might react adversely to the introduced vectors.
Flashcards
What is genetics?
What is genetics?
The branch of biology that studies genes, genetic variation, and heredity in living organisms.
What is a gene?
What is a gene?
A segment of DNA that acts as a blueprint for building and regulating proteins, essential for life.
What is heredity?
What is heredity?
The process by which traits and characteristics are passed from one generation to the next.
What is Mendelian Inheritance?
What is Mendelian Inheritance?
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What causes Genetic Variation?
What causes Genetic Variation?
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What is gene therapy?
What is gene therapy?
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What is the focus of Gene Therapy?
What is the focus of Gene Therapy?
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Why is gene therapy important?
Why is gene therapy important?
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What happened in the 1960s-1970s regarding gene therapy?
What happened in the 1960s-1970s regarding gene therapy?
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What happened in 1980 regarding gene therapy?
What happened in 1980 regarding gene therapy?
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What happened in 2017 regarding gene therapy?
What happened in 2017 regarding gene therapy?
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What is Ex Vivo Gene Therapy?
What is Ex Vivo Gene Therapy?
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What is In Vivo Gene Therapy?
What is In Vivo Gene Therapy?
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What is CRISPR-Cas9 Technology?
What is CRISPR-Cas9 Technology?
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Name some viral vectors in relation to Gene Therapy
Name some viral vectors in relation to Gene Therapy
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What are some Ethical Issues regarding gene therapy?
What are some Ethical Issues regarding gene therapy?
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What are some Safety Concerns regarding gene therapy?
What are some Safety Concerns regarding gene therapy?
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Study Notes
- Gene Therapy is a field relating to Science, Technology and Society
Genetics
- Genetics studies genes, genetic variation, and heredity in living organisms
- A gene is a segment of DNA that acts as a blueprint for building and regulating essential proteins
- Heredity involves traits and characteristic passed from one generation to the next
Inheritance of Traits
- Gregor Mendel's experiments with pea plants showed how traits pass from parents to offspring
- Genetic variation occurs due to mutations, genetic recombination, and independent assortment during reproduction
Genetics and Gene Therapy
- Genetics explains the roles of genes in determining traits and controlling biological processes.
- Gene Therapy directly modifies genes to correct disorders caused by genetic mutations
Details on Gene Therapy
- Gene therapy is a revolutionary medical technique that modifies, replaces, or manipulates genes to treat or prevent diseases
- It addresses the genetic causes of conditions, rather than just the symptoms
- This process replaces faulty or missing genes with functional ones
- It can also inactivate or "silence" a mutated, problematic gene
- It may also introduce a new gene to fight a disease or enhance immunity
Importance of Gene Therapy
- It can offer hope for curing previously untreatable genetic disorders
- It can reduce dependency on lifelong medications or surgeries
- Gene therapy aligns with advancements in personalized medicine, making treatments more specific to an individual's genetic makeup
Historical Background for Gene Therapy
- In the 1960s-1970s, advances in recombinant DNA technology allowed scientists to manipulate genes in laboratories
- In 1980, Dr. Theodore Friedmann and Dr. Richard Roblin proposed gene therapy as a therapeutic approach
- The first FDA-approved gene therapy trial was conducted in 1990 on a 4-year-old girl with Severe Combined Immunodeficiency (SCID)
- CRISPR-Cas9 was introduced in 2000
- Luxturna became the first gene therapy approved by the FDA in 2017 for inherited retinal disease
In Vivo and Ex Vivo Techniques
- In Ex Vivo therapy, cells are removed from the patients body and genetically modified in a controlled laboratory before being reintroduced
- The Ex Vivo process includes extraction, modification, expansion, and reintroduction
- In In Vivo therapy, therapeutic genes are directly delivered into the body, targeting specific cells or tissues
- The In Vivo process includes selection, modification, insertion, and gene expression
Methods of gene delivery
- CRISPR-Cas9 technologies are used for genome editing for precision treatment
- Viral Vectors: Retrovirus, Adenovirus, Lentivirus can be used
Challenges and Limitations
- Gene therapy can have ethical issues with accessibility and inequality in healthcare
- Safety concerns relate to long term side effects
- Technical challenges relate to low efficiency, and immunogenicity of vectors
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