Gene Therapy and Viral Vectors

Gene therapy involves modifying genetic material to treat diseases, often using viral vectors. These vectors, derived from viruses, deliver therapeutic genes into cells. There are four main types: AAVs, adenoviral, lentiviral, and retroviral vectors, each with unique features and uses. AAVs are used for non-dividing cells but face immune challenges. Adenoviral vectors can carry larger genes but initially caused strong immune responses, which have been mitigated. Lentiviral and retroviral vectors integrate into the genome, suitable for ex vivo treatments. Gene therapy is already treating some diseases and is being explored for more applications.

What is the primary purpose of using vectors in gene therapy?

• To create new types of viruses

• To enhance the immune system's response

• To deliver therapeutic genetic material into cells

• To remove harmful genes from the body

Which type of viral vector is typically used for delivering smaller DNA packages?

• Adenoviral vectors

• Lentiviral vectors

• Adeno-associated viral vectors (AAVs)

• Retroviral vectors

What is a limitation of AAVs in gene therapy?

• They integrate into the cell's genome

• They can only be used in dividing cells

• They may be attacked by the immune system if the patient has prior exposure

• They can only deliver RNA

How do adenoviral vectors differ from AAVs in terms of DNA package size?

• They can deliver smaller DNA packages

• They cannot deliver DNA at all

• They can deliver larger DNA packages

• They can only deliver RNA

What was a major challenge with the first generation of adenoviral vectors?

• They were too expensive to produce

• They could not target specific cells

• They caused a strong immune response

• They were too small to carry therapeutic genes

Which vectors are best suited for ex vivo treatment in dividing cells?

• None of the above

• Lentiviral and retroviral vectors

• Adeno-associated viral vectors

• Adenoviral vectors

What is a key feature of lentiviral and retroviral vectors?

• They can only carry small genetic packages

• They do not integrate into the genome

• They integrate into the genome of the target cell

• They are used for in vivo treatments

What is the process called when cells are modified outside the body and then returned?

• Cellular therapy

• Gene editing

• Ex vivo treatment

• In vivo treatment

What diseases are currently being treated with gene therapy using viral vectors?

• Common cold and flu

• Rare inherited diseases and certain cancers

• Allergies and asthma

• Diabetes and hypertension

Where can one find more information about gene therapy and vectors?

• asgct.org

• genetherapy.com

• viralvectors.net

• therapyinfo.org