Retroviral Vectors and Genome Quiz

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Questions and Answers

What is the primary focus of the research mentioned in the text?

Investigating the potential of primordial germ cells to develop into sperm and eggs (correct)

Sequencing the full genome of the Nicobar pigeon

Testing the feasibility of dodo de-extinction

Exploring the applications of gene therapy and cell therapy

What are PGCs, as referred to in the text?

Cells that act as a precursor for ovaries or testes in the Nicobar pigeon (correct)

Intact, living cells delivered to a patient to treat disease

Cells that act as a precursor for ovaries or testes in the dodo

Cells involved in gene therapy and cell therapy

What is gene therapy, as defined in the text?

Delivery of intact, living cells into a patient to treat disease

Sequencing the full genome of a species

The process of replacing, engineering or regenerating human cells, tissues or organs

Delivery of therapeutic gene into a patient's cells to treat disease (correct)

What is cell therapy, according to the text?

The delivery of intact, living cells into a patient to treat disease Signup and view all the answers

What is the relationship between the dodo and the Nicobar pigeon?

The Nicobar pigeon is the dodo’s closest living relative Signup and view all the answers

What is the main purpose of regenerative medicine?

To restore or establish normal function of human cells, tissues or organs Signup and view all the answers

Which type of therapy involves delivering intact, living cells into a patient to treat disease?

Cell therapy Signup and view all the answers

What is the primary goal of gene replacement therapy?

Correcting a cell with a non-functioning gene by introducing a functional gene Signup and view all the answers

Which type of gene therapy involves correcting a cell producing a toxic protein by targeting the abnormal gene to reduce/prevent protein expression?

Gene suppression Signup and view all the answers

What is the main objective of gene editing therapy?

Cutting and repairing DNA to reduce, introduce, or correct the variant Signup and view all the answers

Which type of gene therapy involves removing cells from the body, inserting a gene of interest, culturing the cells, and then returning them to the body?

Ex vivo gene therapy Signup and view all the answers

What are the factors to be considered in gene therapy?

How to deliver genes to specific cells, tissue and whole animals; How much and how long the introduced gene will be expressed; The site and dose of gene delivery Signup and view all the answers

What is the ideal vector for gene transfer?

A vector with high concentration of virus, allowing many cells to be infected or transduced; Convenience and reproducibility of production Signup and view all the answers

Why use viral vectors in gene therapy?

$Virus$ are obligate intracellular parasites; Very efficient at transferring viral DNA into host cells Signup and view all the answers

Which type of virus can infect both actively dividing and non-dividing cells?

Lentiviruses Signup and view all the answers

What is the main risk associated with retrovirus-based gene therapy?

Insertional mutagenesis Signup and view all the answers

Which gene therapy approach involves delivering synthetic DNA containing a normal gene into the immune cells?

Ex vivo gene therapy Signup and view all the answers

What is being used to treat genetic diseases like sickle cell anemia by editing the DNA in bone marrow stem cells?

CRISPR-Cas9 gene-editing system Signup and view all the answers

Which type of gene therapy vectors has the potential for insertional mutagenesis and the need for dividing cells?

Lentiviruses Signup and view all the answers

Which company is focusing on using the CRISPR-Cas9 system to treat genetic diseases?

Vertex and CRISPR Therapeutics Signup and view all the answers

What is used to deliver in vivo gene therapy to the right tissue at the right dose at the right time?

Retroviral vectors Signup and view all the answers

What is the primary concern related to germline gene therapy?

Unknown risks to future generations Signup and view all the answers

What ethical concept is associated with the concern for unknown risks in germline gene therapy?

Safety (nonmaleficence) Signup and view all the answers

What is a potential argument in favor of germline gene therapy?

Eradication of disease in future generations Signup and view all the answers

What concern is associated with the argument against germline gene therapy related to widening the gap between different socioeconomic classes?

Costly use of resources Signup and view all the answers

What reason does W. French Anderson provide in support of germline gene therapy?

Human nature Signup and view all the answers

Which application is listed as another use of recombinant vectors aside from germline gene therapy?

Vaccines Signup and view all the answers

What special topic for group reporting is mentioned in the text?

Current genomics applications available in the Philippines Signup and view all the answers

What argument against germline gene therapy is related to violating genetic integrity of future generations?

Violates genetic integrity of future generations Signup and view all the answers

Study Notes

Retroviruses are a type of virus with a lipid membrane envelope and an ssRNA genome. They can infect both mouse and human cells and integrate randomly into the host genome.
Retroviral vectors are based on Moloney murine leukemia virus (Mo-MLV) and are used to deliver transgenes of interest by replacing the non-essential genes with the transgene.
Lentiviruses are a subtype of retroviruses that can infect both actively dividing and non-dividing cells. They have been used to develop replication incompetent lentiviral vectors for gene therapy.
These vectors are designed by "gutting" the viral genome as much as possible and using a 4-plasmid system. They can only replicate in the presence of the packaging cells and the transfer plasmid.
Retrovirus-based gene therapy carries risks of insertional mutagenesis, which can disrupt endogenous host genes and potentially lead to increased cancer risk. ADA deficiency is one such condition that can be treated with gene therapy, although there is no real cure.
Gene therapy involves delivering synthetic DNA containing a normal gene into the immune cells to restore their function. Successful cases include the treatment of Severe Combined ImmunoDeficiency (SCID) in a 6-year-old girl named Ashanthi DeSilva.
In vivo gene therapy can be delivered to the right tissue at the right dose at the right time using a variety of delivery strategies, including viral vectors and non-viral vectors like liposomes.
Retroviruses, adenoviruses, and adenoassociated viruses are commonly used as gene therapy vectors. Each has its advantages and disadvantages, such as the ability to integrate into chromosomal DNA, the potential for insertional mutagenesis, and the need for dividing cells.
CRISPR-Cas9 gene-editing system is being used to treat genetic diseases like sickle cell anemia by editing the DNA in bone marrow stem cells. This allows the production of normal hemoglobin and can lead to a cure for the disease.
The gene-editing therapy involves transfusions to release bone marrow stem cells into the bloodstream followed by a treatment to edit the genes. Clinical trials have shown positive results, with patients no longer requiring blood transfusions and experiencing a reduction in complications.
Vertex and CRISPR Therapeutics are focusing on using the CRISPR-Cas9 system to treat genetic diseases. The therapy involves editing the genes in bone marrow stem cells to produce normal hemoglobin, which can lead to a cure for sickle cell anemia.
The therapy involves a lengthy process of blood transfusions followed by a treatment to release the bone marrow stem cells into the bloodstream. Clinical trials have shown positive results, with patients no longer requiring blood transfusions and experiencing a reduction in complications.

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Description

Test your knowledge of retroviral vectors and genome with this quiz. Explore concepts such as low titer of virus, host range determination, viral genome components, and more.