W3-2 Clinical Applications of Genome Editing Technology

25 Questions

Which of the following is NOT a learning objective associated with the reading?

Describing the advantages and disadvantages of using viral vectors to deliver genes

What is the critical component of the CRISPR/Cas9 system that makes it the preferred method for genome editing?

Ability to target specific DNA sequences with high precision

Which disease is NOT mentioned as a targeted treatment for genome editing in the reading?

Cystic Fibrosis

What cellular process is essential for efficient gene therapy?

Transfection

What is the main advantage of using gene editing over enzyme replacement therapy (ERT) and gene therapy in clinical medicine today?

Ability to correct genetic mutations at the DNA level

What distinguishes gene knock-in from gene knock-out?

Gene knock-in adds a specific gene, while gene knock-out deactivates a specific gene.

What is the main function of TALENs?

DNA recognition

Which method requires a homologous template DNA for repair?

Homologous repair

What is the function of the protospacer adjacent motif (PAM) in CRISPR systems?

Required for Cas nuclease to cut

What is the abbreviation sgRNA stand for and why?

Short guide RNA, because it is only up to 100 bp

What is the primary cause of sickle-cell anemia?

A to T transversion in the second nucleotide of codon 6

What does the CRISPR/Cas9 method utilize to make a double-stranded cut in DNA?

sgRNA

What is the role of micro homologous end joining?

Provides a short DNA sequence for Cas nuclease to cut

Which method eliminates the need for host recombination?

CAST transposase

"Knock out" refers to which type of genetic modification?

"Disrupting a gene (artificial loss-of-function mutation)"

"Knock in" is used for which type of genetic modification?

"Changing a gene into a different version"

Which of the following is NOT a goal of gene therapy?

Get anti-inflammatory genes into joints of arthritis patients

What is the main challenge in gene therapy related to getting foreign DNA into the cell?

Cytotoxicity due to copy number or integration locations

How does a retrovirus differ from other viruses?

It integrates its RNA genome into the DNA of a host cell

What is the function of Elaprase in the treatment of severe mucopolysaccharidosis?

Recombinant enzyme for breaking down glycosaminoglycans

Which virus vector has been FDA approved for treating retinal dystrophy?

Adenovirus-associated virus vectors

What is the primary purpose of stem cell replacement therapy?

Replacement of defective cells with altered cells grown throughout the patient's life

What technique is currently used as the method of choice for genome editing in gene therapy?

CRISPR/Cas9

What is the purpose of FLT180a gene therapy in patients with hemophilia B?

To normalize factor IX levels by using a synthetic capsid and gain-of-function protein

What are the common genetic diseases addressed by gene therapy?

(MPS I, MPS II, MPS IVa or MPS VI)

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.

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.

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