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Questions and Answers
Which of the following is NOT a learning objective associated with the reading?
Which of the following is NOT a learning objective associated with the reading?
What is the critical component of the CRISPR/Cas9 system that makes it the preferred method for genome editing?
What is the critical component of the CRISPR/Cas9 system that makes it the preferred method for genome editing?
Which disease is NOT mentioned as a targeted treatment for genome editing in the reading?
Which disease is NOT mentioned as a targeted treatment for genome editing in the reading?
What cellular process is essential for efficient gene therapy?
What cellular process is essential for efficient gene therapy?
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What is the main advantage of using gene editing over enzyme replacement therapy (ERT) and gene therapy in clinical medicine today?
What is the main advantage of using gene editing over enzyme replacement therapy (ERT) and gene therapy in clinical medicine today?
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What distinguishes gene knock-in from gene knock-out?
What distinguishes gene knock-in from gene knock-out?
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What is the main function of TALENs?
What is the main function of TALENs?
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Which method requires a homologous template DNA for repair?
Which method requires a homologous template DNA for repair?
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What is the function of the protospacer adjacent motif (PAM) in CRISPR systems?
What is the function of the protospacer adjacent motif (PAM) in CRISPR systems?
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What is the abbreviation sgRNA stand for and why?
What is the abbreviation sgRNA stand for and why?
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What is the primary cause of sickle-cell anemia?
What is the primary cause of sickle-cell anemia?
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What does the CRISPR/Cas9 method utilize to make a double-stranded cut in DNA?
What does the CRISPR/Cas9 method utilize to make a double-stranded cut in DNA?
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What is the role of micro homologous end joining?
What is the role of micro homologous end joining?
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Which method eliminates the need for host recombination?
Which method eliminates the need for host recombination?
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"Knock out" refers to which type of genetic modification?
"Knock out" refers to which type of genetic modification?
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"Knock in" is used for which type of genetic modification?
"Knock in" is used for which type of genetic modification?
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Which of the following is NOT a goal of gene therapy?
Which of the following is NOT a goal of gene therapy?
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What is the main challenge in gene therapy related to getting foreign DNA into the cell?
What is the main challenge in gene therapy related to getting foreign DNA into the cell?
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How does a retrovirus differ from other viruses?
How does a retrovirus differ from other viruses?
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What is the function of Elaprase in the treatment of severe mucopolysaccharidosis?
What is the function of Elaprase in the treatment of severe mucopolysaccharidosis?
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Which virus vector has been FDA approved for treating retinal dystrophy?
Which virus vector has been FDA approved for treating retinal dystrophy?
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What is the primary purpose of stem cell replacement therapy?
What is the primary purpose of stem cell replacement therapy?
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What technique is currently used as the method of choice for genome editing in gene therapy?
What technique is currently used as the method of choice for genome editing in gene therapy?
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What is the purpose of FLT180a gene therapy in patients with hemophilia B?
What is the purpose of FLT180a gene therapy in patients with hemophilia B?
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What are the common genetic diseases addressed by gene therapy?
What are the common genetic diseases addressed by gene therapy?
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Study Notes
- Dr. Zahi Damuni is a professor of Biochemistry with expertise in genome editing technology.
- The text covers learning objectives, guiding questions, and therapies related to enzyme replacement therapy (ERT), gene therapy, gene editing, and their applications in clinical medicine.
- ERT involves infusing missing or defective proteins into patients routinely. Conditions such as hemophilia A, B, and various metabolic diseases benefit from ERT.
- Gene therapy aims to insert a functional copy of a defective gene into the cells of a patient, treating diseases like sickle cell, muscular dystrophy, and cancer.
- Gene editing uses techniques like CRISPR/Cas9 to modify or replace specific genes, with applications in biomedical research and medicine.
- Hemophilia A and B have seen recent advances in treatment through gene therapy using viral vectors like AAV5–Factor VIII Gene Transfer and AAVS3 Gene Therapy.
- Sickle Cell Gene Therapy is an emerging treatment where a patient's own cells are modified and reintroduced into their body.
- Enzyme replacement therapy, gene therapy, and gene editing are distinguished by their mechanisms and therapeutic approaches.
- Viral vectors used in gene therapy may integrate randomly into the genome or only transiently infect cells, causing challenges in delivering effective treatments.
- CRISPR/Cas9 is the most commonly used gene editing system due to its high precision and efficiency.
- Gene therapy using virus vectors has faced challenges like random integration, transient infection, cytotoxicity, and limited application to certain cells.
- CRISPR/Cas9 uses a small guide RNA to target a specific sequence in DNA and induce a double-stranded break, allowing for precise modifications.
- The text also discusses the importance of the protospacer adjacent motif (PAM) in the CRISPR/Cas9 system and how it aids in DNA cleavage.
- ERT, gene therapy, and gene editing have their unique advantages and limitations, and their clinical applications continue to evolve in treating various diseases.
- Haemophilia A and B have seen significant advancements in treatment through gene therapy using viral vectors like AAV5–Factor VIII Gene Transfer and AAVS3 Gene Therapy.
- Sickle Cell Gene Therapy is an emerging treatment where a patient's own cells are modified and reintroduced into their body.
- Enzyme replacement therapy, gene therapy, and gene editing are distinguished by their mechanisms and therapeutic approaches.
- Viral vectors used in gene therapy may integrate randomly into the genome or only transiently infect cells, causing challenges in delivering effective treatments.
- CRISPR/Cas9 is the most commonly used gene editing system due to its high precision and efficiency.
- Gene therapy using virus vectors has faced challenges like random integration, transient infection, cytotoxicity, and limited application to certain cells.
- CRISPR/Cas9 uses a small guide RNA to target a specific sequence in DNA and induce a double-stranded break, allowing for precise modifications.
- The text also discusses the importance of the protospacer adjacent motif (PAM) in the CRISPR/Cas9 system and how it aids in DNA cleavage.
- ERT, gene therapy, and gene editing have their unique advantages and limitations, and their clinical applications continue to evolve in treating various diseases.
- CRISPR/Cas9 is a powerful tool for genome editing, with applications in both research and medicine.
- Gene editing methods like CRISPR/Cas9 require endonucleases to cut the genome, which then triggers the cell's repair mechanisms.
- Precise genome editing using CRISPR/Cas9 involves injecting the DNA endonuclease into a cell and using the cell's own repair systems to incorporate the desired modifications.
- Non-homologous end joining and homologous repair are the primary pathways for DNA repair in response to double-strand breaks induced by CRISPR/Cas9.
- CRISPR/Cas9 relies on a protospacer adjacent motif (PAM) to recognize and target specific sequences in the genome.
- sgRNA is a small RNA molecule used in the CRISPR/Cas9 system to guide the Cas9 enzyme to the target sequence in the genome.
- CRISPR/Cas9 uses the cell's recombination systems to incorporate the desired modifications into the genome, making it a highly versatile and efficient gene editing tool.
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Description
Test your knowledge of genome editing technology and its clinical applications with this quiz. Learn about enzyme replacement therapy, gene therapy, CRISPR/Cas9 system, and its critical components.