Podcast
Questions and Answers
What is the primary outcome of germinal genetic modification?
What is the primary outcome of germinal genetic modification?
- Temporary changes in somatic cells
- No changes to the genome
- Permanent changes in reproductive cells (correct)
- Reversible modifications that can be cured
What major ethical concern was raised in the Jiankui case regarding the gene editing of embryos?
What major ethical concern was raised in the Jiankui case regarding the gene editing of embryos?
- The research was conducted with full parental consent
- It involved enhancing a genetic trait rather than treating a disease (correct)
- The process was thoroughly peer-reviewed
- The experiment had a clear therapeutic benefit
Which DNA repair mechanism is associated with random insertions or deletions that can lead to mutations?
Which DNA repair mechanism is associated with random insertions or deletions that can lead to mutations?
- Nonhomologous end-joining (correct)
- Nucleotide excision repair
- Homology-directed repair
- Base excision repair
What was the significance of the 2018 event involving the twins Lulu and Nana?
What was the significance of the 2018 event involving the twins Lulu and Nana?
What main risk is associated with gene editing that results in mosaicism?
What main risk is associated with gene editing that results in mosaicism?
Which gene was targeted in the Jiankui case for modification related to HIV protection?
Which gene was targeted in the Jiankui case for modification related to HIV protection?
What is the main criticism regarding the benefit-risk balance of Jiankui's experiment?
What is the main criticism regarding the benefit-risk balance of Jiankui's experiment?
Which of the following statements about homology-directed repair (HDR) is accurate?
Which of the following statements about homology-directed repair (HDR) is accurate?
What type of enzyme is an endonuclease?
What type of enzyme is an endonuclease?
What does CRISPR stand for?
What does CRISPR stand for?
Which gene editing tool is characterized as being more difficult and expensive than CRISPR?
Which gene editing tool is characterized as being more difficult and expensive than CRISPR?
Who were the key figures in elucidating the mechanism of CRISPR/Cas9 in 2012?
Who were the key figures in elucidating the mechanism of CRISPR/Cas9 in 2012?
What are the three phases of the natural CRISPR system?
What are the three phases of the natural CRISPR system?
What is the purpose of the trial evaluating EDIT-101?
What is the purpose of the trial evaluating EDIT-101?
What gene is associated with Leber congenital amaurosis 10 (LCA10)?
What gene is associated with Leber congenital amaurosis 10 (LCA10)?
Which experimental treatment showed promise for an inherited form of deafness?
Which experimental treatment showed promise for an inherited form of deafness?
What is the significance of the case of Aissam Dam?
What is the significance of the case of Aissam Dam?
What biotechnological concern was raised regarding CRISPR technology?
What biotechnological concern was raised regarding CRISPR technology?
What was the outcome of the PRRS virus inoculation in genetically modified piglets?
What was the outcome of the PRRS virus inoculation in genetically modified piglets?
What was the primary goal of the clinical trials mentioned in the context of gene therapy for deafness?
What was the primary goal of the clinical trials mentioned in the context of gene therapy for deafness?
Which statement about the first experimental programs in gene therapies is true?
Which statement about the first experimental programs in gene therapies is true?
Which crop is made resistant to powdery mildew disease through gene editing?
Which crop is made resistant to powdery mildew disease through gene editing?
Which gene is targeted to confer resistance to bacterial blight in rice?
Which gene is targeted to confer resistance to bacterial blight in rice?
What type of changes does gene editing primarily introduce into crops?
What type of changes does gene editing primarily introduce into crops?
In the context of regulations, gene-edited crops in the European Union are treated the same as which of the following?
In the context of regulations, gene-edited crops in the European Union are treated the same as which of the following?
Which disease is eliminated in Cucumis sativus L through targeted gene editing?
Which disease is eliminated in Cucumis sativus L through targeted gene editing?
What distinguishes gene editing from traditional GMOs?
What distinguishes gene editing from traditional GMOs?
Which disease is associated with Arabidopsis thaliana?
Which disease is associated with Arabidopsis thaliana?
Which of the following crops is treated to develop resistance against the leaf thickening disease?
Which of the following crops is treated to develop resistance against the leaf thickening disease?
What will occur if one parent carries two disease-causing alleles?
What will occur if one parent carries two disease-causing alleles?
What is true about expectant mothers who carry two disease-causing alleles?
What is true about expectant mothers who carry two disease-causing alleles?
What is a characteristic of X-linked recessive diseases?
What is a characteristic of X-linked recessive diseases?
Which statement reflects the ethical concerns of somatic gene editing (GE)?
Which statement reflects the ethical concerns of somatic gene editing (GE)?
What is a possible outcome if both parents carry two disease-causing alleles?
What is a possible outcome if both parents carry two disease-causing alleles?
Why might research efforts be directed away from certain genetic disease studies?
Why might research efforts be directed away from certain genetic disease studies?
What determines whether male offspring are affected by X-linked recessive diseases?
What determines whether male offspring are affected by X-linked recessive diseases?
What does the summit recommend regarding germline gene editing at the current time?
What does the summit recommend regarding germline gene editing at the current time?
What is the general stance of the scientific community on somatic gene editing?
What is the general stance of the scientific community on somatic gene editing?
What kind of therapy did Layla Richards receive for her leukemia?
What kind of therapy did Layla Richards receive for her leukemia?
What was a notable outcome of the treatment received by Layla Richards?
What was a notable outcome of the treatment received by Layla Richards?
Which of the following statements about the current state of somatic genetic modification is true?
Which of the following statements about the current state of somatic genetic modification is true?
What is the primary function of the CAR-T cell therapy in treating cancer?
What is the primary function of the CAR-T cell therapy in treating cancer?
What was the initial condition of Layla Richards at 14 weeks of age?
What was the initial condition of Layla Richards at 14 weeks of age?
How does the summit describe the current understanding of the scientific and technical requirements for germline gene editing?
How does the summit describe the current understanding of the scientific and technical requirements for germline gene editing?
What is a key characteristic of UCART19 cells as described in the content?
What is a key characteristic of UCART19 cells as described in the content?
Flashcards
What is gene editing?
What is gene editing?
Gene editing is a powerful technology that allows scientists to precisely modify the DNA sequence of an organism. These alterations can range from correcting disease-causing mutations to introducing beneficial traits. Gene editing tools, like CRISPR-Cas, are revolutionizing various fields, including medicine, agriculture, and biotechnology.
What is Recombinant DNA technology?
What is Recombinant DNA technology?
Recombinant DNA technology involves combining DNA from different sources to create new genetic combinations. This is achieved by cutting and pasting DNA fragments using restriction enzymes and ligases, allowing the creation of novel molecules with potential applications in medicine and agriculture.
What is CRISPR-Cas?
What is CRISPR-Cas?
CRISPR-Cas is a gene editing tool derived from a natural defense system found in bacteria and archaea. It consists of a protein (Cas) enzyme and a guide RNA (gRNA) that can target and cut specific DNA sequences. This targeted cutting allows for the insertion, deletion, or modification of genes, enabling precise genome editing.
What are ZFNs and TALENs?
What are ZFNs and TALENs?
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Who are the key figures in CRISPR-Cas?
Who are the key figures in CRISPR-Cas?
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Autosomal dominant disease
Autosomal dominant disease
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Autosomal recessive disease
Autosomal recessive disease
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X-linked recessive disease
X-linked recessive disease
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Somatic Gene Editing (SGE)
Somatic Gene Editing (SGE)
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Germline Gene Editing (GGE)
Germline Gene Editing (GGE)
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Gene editing for treating genetic diseases
Gene editing for treating genetic diseases
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Current position on SGE
Current position on SGE
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Ethical considerations in SGE
Ethical considerations in SGE
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Nonhomologous End-Joining (NHEJ)
Nonhomologous End-Joining (NHEJ)
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Homology-Directed Repair (HDR)
Homology-Directed Repair (HDR)
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Germline Genetic Modification
Germline Genetic Modification
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NHEJ in DNA Repair
NHEJ in DNA Repair
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Mosaicism
Mosaicism
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Off-target Effects
Off-target Effects
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Genetic Enhancement
Genetic Enhancement
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Human Germline Modification
Human Germline Modification
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Somatic genetic modification
Somatic genetic modification
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CAR-T cell therapy
CAR-T cell therapy
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TALENs
TALENs
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Layla Richards
Layla Richards
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Germline vs. somatic editing
Germline vs. somatic editing
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2018 International Summit on Human Genome Editing
2018 International Summit on Human Genome Editing
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Future of gene editing
Future of gene editing
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Ethical considerations of gene editing
Ethical considerations of gene editing
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EDIT-101
EDIT-101
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Leber Congenital Amaurosis 10 (LCA10)
Leber Congenital Amaurosis 10 (LCA10)
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Otoferlin gene
Otoferlin gene
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Gene therapy for deafness
Gene therapy for deafness
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CRISPR technology
CRISPR technology
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CRISPR-edited livestock
CRISPR-edited livestock
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Porcine Reproductive and Respiratory Syndrome (PRRS)
Porcine Reproductive and Respiratory Syndrome (PRRS)
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Sickle Cell Disease
Sickle Cell Disease
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CRISPR-Cas9 in Crop Disease Resistance
CRISPR-Cas9 in Crop Disease Resistance
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How does CRISPR-Cas9 work?
How does CRISPR-Cas9 work?
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TaMLO-A1 Gene in Wheat
TaMLO-A1 Gene in Wheat
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OsSWEET11 and OsSWEET14 in Rice
OsSWEET11 and OsSWEET14 in Rice
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CRISPR-Cas9 vs. GMOs
CRISPR-Cas9 vs. GMOs
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EU Regulation on CRISPR-Cas9 Crops
EU Regulation on CRISPR-Cas9 Crops
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Applications of CRISPR-Cas9 in Crop Improvement
Applications of CRISPR-Cas9 in Crop Improvement
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Random Mutagenesis vs. CRISPR-Cas9
Random Mutagenesis vs. CRISPR-Cas9
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Study Notes
Unit 4: Gene Editing. CRISPR
- Gene editing modifies the genome, contexts (epigenetic marks), or results (transcripts) using endonucleases, enzymes that cut DNA.
Previous Technology: Recombinant DNA
- Recombinant DNA technology involves inserting a gene of interest into a plasmid, placing the plasmid into a bacterium, and growing the bacterium in a culture to create copies of the gene.
- The resulting protein can be harvested and used in research and certain applications (e.g., pest resistance crops, treating diseases).
Genetic Editing
- Recombinant DNA is a previous technology compared to gene editing.
- Gene editing, using newer techniques like CRISPR, has greater precision and lower costs than recombinant DNA techniques.
- Inaccuracies, ineffectiveness, and applicability factors are potential problems in gene editing.
Gene Editing Tools
- ZFNs (Zinc Finger Nucleases): High cost, complex operation, and operating problems.
- TALENs (Transcription Activator-Like Effector Nucleases): More challenging and expensive compared to CRISPR.
- CRISPR-Cas: Easier and more affordable technology that has revolutionized this field.
CRISPR-Cas
- A natural defense system in bacteria and archaea against viruses.
- CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats): Repeating DNA sequences separated by short segments of foreign DNA, important component of the system.
- Cas (CRISPR-associated): Proteins that form a complex with crRNA and work together to cut foreign DNA at specific locations.
The Natural System (CRISPR-Cas)
- A 3-step process for defending against viruses in prokaryotic cells:
- Acquisition: Foreign viral DNA is incorporated into CRISPR locus.
- Expression: CRISPR RNA (crRNA) is processed from the CRISPR locus and forms a complex with Cas proteins.
- Interference: The crRNA-Cas complex targets and degrades matching viral DNA, preventing infection.
CRISPR as a Gene Editing Tool
- Timeline of significant milestones in CRISPR development, research, and application.
- In 1987, CRISPR was first reported.
- Between 2000 and 2014, discoveries about CRISPR-Cas systems and their applications in various fields emerged.
- In 2012, Charpentier and Doudna revealed the mechanism of CRISPR/Cas9 and demonstrated its potential for gene editing.
- The Nobel Prize in Chemistry 2020 was granted to Emmanuelle Charpentier and Jennifer A. Doudna for discovering this method.
DNA Repair Mechanisms
- Non-homologous end joining (NHEJ): A fast repair mechanism, but prone to errors like insertions or deletions.
- Homology-directed repair (HDR): A more precise repair pathway where a template is used to repair the DNA break.
###Applications of CRISPR
- Gene therapy has promising potential
- Applications in medicine, including drug development, animal models, gene surgery, diagnostics, and materials science, as well as biotechnology.
- Uses in agriculture (e.g., crops resistant to diseases and environmental factors).
- Germline genetic modification affects reproductive cells (sperm and egg) or early embryonic development and results in heritable changes to future generations.
Medical Applications
- Somatic genetic modification targets non-reproductive cells (like stem cells) for treating diseases such as cancer and genetic disorders.
- Germline modification targets reproductive cells and early embryos.
CRISPR Case Examples
- Layla Richards received treatment for leukemia using CAR-T cell therapy which was based on a newer gene editing technology (TALENs).
- A woman with sickle cell disease was cured using CRISPR gene editing to change a specific gene.
Risks and Ethical Considerations Associated with CRISPR
- Off-target effects
- Mosaicism
- Risks associated with genome editing, including potential for unintended mutations and safety/ethical issues.
- Ethical issues related to using CRISPR on human germline cells for modifications that will affect future generations of individuals.
- Unfavorable benefit-risk balance, and falsified ethical documents.
- Potential risks to the ecosystem, food chain, and appearance of new pests.
- The possibility of the spread of mutated guide RNAs, and dual use technology of the method itself
CRISPR-Edited Plants
- Specific examples of plant varieties or crops altered to be resistant to environmental stressors or diseases.
Crops Resistant to Diseases
- Specific examples of crop varieties or rice that have been made resistant to disease using CRISPR/Cas9.
- Some crops have been genetically modified to resist diseases (e.g., rice with enhanced disease resistance).
Transgenic Organisms vs. Gene Editing
- Differentiate between using foreign genes (transgenic) vs. making small alterations in existing genes (gene editing).
- How they apply to organisms.
EU Regulations
- Restrictions on the use of CRISPR in plant gene editing.
- Research in the EU regarding genome editing.
Safety and Additional Notes
- Safety and ethical aspects of CRISPR.
- Need for lengthy analysis of characteristics when testing and selecting gene-edited organisms.
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