Gene therapy paradigms

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Help the frog cross the river by designing the most suitable gene therapies!

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Maitreyi

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Froggy Jumps Gene therapy paradigmsVersión en línea Help the frog cross the river by designing the most suitable gene therapies! por Maitreyi 1 Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. Which of the following paradigms would you choose? a Increase gene expression b Decreased gene expression c Induce cell death 2 Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to increase gene expression. Which of these vectors will you use? a Adenoviral vector b Herpes simplex viral vector c Retroviral vector 3 Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to use a retroviral vector. How will you administer the gene therapy? a Intravenous b Intravitreal injection c Intramuscular injection 4 Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which paradigm would you choose? a Increase gene expression b Decrease gene expression c Induce cell death 5 Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which mechanisms is most suitable? a Gene augmentation b Exon skipping c RNAi 6 Condition B is caused by mutations in a large multi-exon gene. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. You want to use exon skipping. Which viral vector is most suitable? a Adeno-associated viral vector b Herpes simplex viral vector c Adenoviral vector

Froggy Jumps

Gene therapy paradigmsVersión en línea

Help the frog cross the river by designing the most suitable gene therapies!

por Maitreyi

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. Which of the following paradigms would you choose?

Increase gene expression

Decreased gene expression

Induce cell death

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to increase gene expression. Which of these vectors will you use?

Adenoviral vector

Herpes simplex viral vector

Retroviral vector

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to use a retroviral vector. How will you administer the gene therapy?

Intravenous

Intravitreal injection

Intramuscular injection

Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which paradigm would you choose?

Increase gene expression

Decrease gene expression

Induce cell death

Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which mechanisms is most suitable?

Gene augmentation

Exon skipping

RNAi

Condition B is caused by mutations in a large multi-exon gene. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. You want to use exon skipping. Which viral vector is most suitable?

Adeno-associated viral vector

Herpes simplex viral vector

Adenoviral vector

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Gene therapy paradigms

Froggy Jumps

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Gene therapy paradigmsVersión en línea

por Maitreyi

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. Which of the following paradigms would you choose?

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to increase gene expression. Which of these vectors will you use?

Condition A is an autosomal recessive disease that results in blindness due to absence of enzymatic activity in the eye. The coding region of the enzyme is 5 kb. You want to use a retroviral vector. How will you administer the gene therapy?

Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which paradigm would you choose?

Condition B is caused by mutations in a large multi-exon gene, which causes the protein not to be functional. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. Which mechanisms is most suitable?

Condition B is caused by mutations in a large multi-exon gene. The mutations are in repetitive structural regions of the gene. The gene is >200,000 base pairs long. You want to use exon skipping. Which viral vector is most suitable?

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