AAV Immune Response and Possible Therapies for Various Diseases

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12 Questions

Which characteristic of the Vaccinia virus genome is mentioned in the text?

Linear, double-stranded DNA

What is the primary location for replication of the Vaccinia virus within the host cell?

Cytoplasm

Which of the following diseases is caused by the Variola virus?

Smallpox

What is the primary characteristic that differentiates Poxviridae from other viruses?

Having a brick-shaped appearance

In AAV vectors, what is the significance of Inverted Terminal Repeats (ITRs)?

They serve as a site for integration into the host chromosome

What is the primary humoral immune response responsible for regarding AAV vectors?

Neutralization of AAV vectors

Which chromosome does the adeno-associated virus (AAV) genome integrate into during site-specific integration?

Chromosome 19q13.4

What are the sequences at the ends of the AAV genome called, and what is their function?

Inverted terminal repeats (ITRs), required for efficient multiplication and integration of the AAV genome

What are the two open reading frames (ORFs) present in the AAV genome, and what are their functions?

Rep gene (regulates AAV gene expression) and Cap gene (encodes viral capsid proteins)

What is the primary difference between recombinant AAV (rAAV) vectors and self-complementary (scAAV) recombinant vectors?

rAAV vectors require second-strand synthesis before gene expression, while scAAV vectors have a shorter lag time

What is the primary function of the Rep gene in the AAV genome?

Regulates AAV gene expression and DNA replication

How are the positive (+) and negative (-) strands of the AAV genome packaged into the viral capsid?

Both strands are packaged with equal frequency

Explore the humoral immune response to AAV vectors and potential therapies for over 50 diseases, including autoimmune arthritis, infectious diseases, metabolic disorders, neurology conditions, neuromuscular disorders, ophthalmological conditions, and pulmonary diseases.

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