Gene Therapy

Question 1

Advantages of hematopoietic stem cells in gene therapy

Answer 1

Precursors for all immune cells: ability to differentiate and expand
Correction of a small number of cells ensure correction of many more cells long-term
Good for treating immunodeficiencies

Question 2

X-linked severe combined immunodeficiency

Answer 2

Can be corrected by gene therapy

Replacement of gamma c receptor (cytokine receptor) treats immunodeficiency in B, T, and NK cells

Question 3

ADA-severe combined immunodeficiency

Answer 3

Can be corrected by gene therapy

Replacement of adenosine deaminase (ADA) corrects defect in B, T, and NK cells

Question 4

Beta-thalassemia

Answer 4

Can be corrected by gene therapy

Replacement of beta-globin gene

Question 5

Leukemia

Answer 5

Can be corrected by gene therapy

Anti-CD19 CAR gene in T cells: binding of tumor CD19 to modified T cells activates immune response

Question 6

Leber’s congenital amaurosis

Answer 6

Can be corrected by gene therapy

Replace retinal protein, correcting blindness

Question 7

Spinal muscular atrophy

Answer 7

Can be corrected by gene therapy

Replacement of SMN1 gene lengthens life and enables children with disease to meet developmental milestones

Question 8

Hemophilia B

Answer 8

Can be corrected by gene therapy

Replacement of factor IX: lessened need for infusion

Question 9

Mucopolysaccharidosis type I

Answer 9

Can be corrected by gene therapy

Replacement of alpha-L-iduronidase decreases glycosaminoglycan levels and prevents neurocognitive deficits

Question 10

Mucopolysaccharidosis type II

Answer 10

Can be corrected by gene therapy

Replacement of iduronate 2 sulfatase