Stem Cells V

Question 1

What are some exogenous manipulations for SC-based therapeutic strategy?

Answer 1

SC isolation and propagation by transplantation or differentiation modification

Question 2

What are some endogenous manipulations for SC-based therapeutic strategy?

Answer 2

Renewal or differentiation of SCs
Activation or inhibition of niche

Question 3

Mesenchymal stem cells are derived from where?

Answer 3

Bone marrow stroma

Question 4

Mesenchymal stem cells can produced what types of tissues?

Answer 4

Bone, cartilage, tendon, adipocyte, muscle, stroma

Question 5

What approach is used to fill the gaps from bone fractures?

Answer 5

Implantation of ceramics cylinder loaded with MSCs

Question 6

What is an approach to regenerative therapy using stem cells?

Answer 6

Engineering of stem cell niches

Question 7

Matrix __________ controls stem cell fate

Answer 7

Elasticity

Question 8

Cell-type-specific genes are differentially expressed depending on ___________

Answer 8

Elasticity

Question 9

Between neurogenic, myogenic and osteogenic cells, which are more elastic?

Answer 9

Osteogenic

Question 10

Sphere culture system is applicable for drug _____________

Answer 10

Screening

Question 11

Which cells can be used for drug discovery?

Answer 11

Neurospheres

Question 12

What are two ways to produce lead compounds for drug discovery?

Answer 12

Fragment-based screening and diversity-oriented synthesis

Question 13

Therapy based on stem cells targets the cause of (pathology/symptoms)

Answer 13

Pathology

Question 14

What are the advantages of using retroviral vectors?

Answer 14

Efficient, works in vivo and in vitro, targets many cells at once, can be permanent

Question 15

Which technique is used for gene editing?

Answer 15

CRISPR/Cas9

Question 16

What can one do with CRISPR/Cas9?

Answer 16

Introducing insertion/deletion mutations, replacing one sequence with another, adding a new sequence, manipulating RNAs

Question 17

What would be the therapeutic strategy for anemia?

Answer 17

Harvest HSCs, interrogate the HSC genome and correct mutation in globin gene ex vivo, transfer modified HSCs back to patient, HSCs engraft and produce RBCs with normal globins

Question 18

Gene editing in HSCs allows for ___________ expression in erythrocytes in adult patients

Answer 18

Gamma-globin (fetal globin)

Question 19

What induces fetal globin expression in adult patients?

Answer 19

BCL11A knockout

Question 20

What is expected from disrupting GATA-binding sequence in the BCL11A enhancer using CRISPR/Cas9?

Answer 20

Reduce BCL11A expression, reactivate gamma-globin expression