Gene Therapy Quiz: Test Your Knowledge of Gene Therapy

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Questions and Answers

What is the main goal of gene replacement?

To kill products or inhibit growth of cells

To affect neighboring and distal tissues

To replace defective/missing product (correct)

To affect the cell itself

Which type of therapy involves the delivery of intact living cells?

Cell therapy (correct)

Gene replacement

Adenoviral therapy

Ex vivo

What is the ideal vector for gene transfer typically?

Fungus

Bacteria

Virus (correct)

Parasite

What is the main goal of gene editing?

To repair a DNA variant Signup and view all the answers

What is the method of delivery for ex vivo therapy?

Cells are removed, modified in lab, and re-inserted Signup and view all the answers

Which type of vector is useful for treating genetic disorders?

Adenoviral Signup and view all the answers

What is the purpose of gene suppression?

To prevent expression of genes Signup and view all the answers

What does in vivo therapy involve?

Introduced directly into cells Signup and view all the answers

What should be considered for gene therapy in terms of site and dose?

'Site and dose' Signup and view all the answers

Which vector has the ability to integrate into a site-specific location?

Adenoviral Signup and view all the answers

Which type of cell therapy involves cells being removed, modified in a lab, and then re-introduced into the body?

Ex vivo Signup and view all the answers

What determines the transient or permanence of expression in gene therapy?

Method of delivery Signup and view all the answers

What is the main purpose of lentivirus in gene therapy?

To deliver insert to the genome Signup and view all the answers

What is a potential risk associated with retroviral gene therapy?

Random insertional mutagenesis Signup and view all the answers

What is a characteristic of 3rd generation lentiviruses used in gene therapy?

Employ a 3-plasmid system for packaging Signup and view all the answers

What is a concern related to the use of gene therapies in general?

Unique to each individual Signup and view all the answers

What is a disadvantage of monoclonal antibodies (mAbs)?

Genetic drift due to prolonged culture passaging Signup and view all the answers

What is the main application of Western blot using antibodies?

Cell and tissue labeling Signup and view all the answers

What is the primary obstacle in using monoclonal antibodies (mAbs) for cancer treatment?

Homogeneous tumor cells with similar antigenic determinants Signup and view all the answers

What is the main target of Trastuzumab (Herceptin) in cancer treatment?

HER2 receptor overexpressed in breast cancer Signup and view all the answers

Gemtuzumab (Mylotarg) is a monoclonal antibody conjugated to what cytotoxic agent for treating acute myelogenous leukemia?

Calicheamicin Signup and view all the answers

Study Notes

Gene Therapy

The main goal of gene replacement is to replace a faulty or missing gene with a healthy copy.
The main goal of gene editing is to correct a specific mutation or defect in a gene.
Gene suppression is used to reduce or eliminate the expression of a specific gene.

Gene Transfer

The ideal vector for gene transfer is typically a viral vector, as it can efficiently deliver genes to target cells.
Lentivirus is a type of vector that can integrate into a site-specific location.
Retroviral vectors are useful for treating genetic disorders, but may have a potential risk of insertional mutagenesis.

Cell Therapy

Ex vivo therapy involves removing cells from the body, modifying them in a lab, and then re-introducing them into the body.
This type of cell therapy is often used in gene therapy to modify cells before re-introducing them into the body.

Vector Characteristics

The ability of a vector to integrate into a site-specific location determines the transient or permanence of expression in gene therapy.
3rd generation lentiviruses are designed to be safer and more efficient, with a characteristic of having a self-inactivating (SIN) design.

Gene Therapy Concerns

A concern related to the use of gene therapies in general is the potential for off-target effects or unintended consequences.

Monoclonal Antibodies (mAbs)

A disadvantage of monoclonal antibodies (mAbs) is that they may not be able to penetrate solid tumors.
The primary obstacle in using monoclonal antibodies (mAbs) for cancer treatment is the development of resistance.
The main application of Western blot using antibodies is to detect and analyze specific proteins in a sample.
Trastuzumab (Herceptin) is a monoclonal antibody that targets the HER2 protein in cancer treatment.
Gemtuzumab (Mylotarg) is a monoclonal antibody conjugated to the cytotoxic agent calicheamicin, used to treat acute myelogenous leukemia.

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Description

This quiz covers the branches of regenerative medicine, including gene therapy and cell therapy, the types of gene therapy, goals of gene therapy, and different kinds of therapies such as ex vivo and in vivo.