Stem Cells V Flashcards
What are some exogenous manipulations for SC-based therapeutic strategy?
SC isolation and propagation by transplantation or differentiation modification
What are some endogenous manipulations for SC-based therapeutic strategy?
Renewal or differentiation of SCs
Activation or inhibition of niche
Mesenchymal stem cells are derived from where?
Bone marrow stroma
Mesenchymal stem cells can produced what types of tissues?
Bone, cartilage, tendon, adipocyte, muscle, stroma
What approach is used to fill the gaps from bone fractures?
Implantation of ceramics cylinder loaded with MSCs
What is an approach to regenerative therapy using stem cells?
Engineering of stem cell niches
Matrix __________ controls stem cell fate
Elasticity
Cell-type-specific genes are differentially expressed depending on ___________
Elasticity
Between neurogenic, myogenic and osteogenic cells, which are more elastic?
Osteogenic
Sphere culture system is applicable for drug _____________
Screening
Which cells can be used for drug discovery?
Neurospheres
What are two ways to produce lead compounds for drug discovery?
Fragment-based screening and diversity-oriented synthesis
Therapy based on stem cells targets the cause of (pathology/symptoms)
Pathology
What are the advantages of using retroviral vectors?
Efficient, works in vivo and in vitro, targets many cells at once, can be permanent
Which technique is used for gene editing?
CRISPR/Cas9
What can one do with CRISPR/Cas9?
Introducing insertion/deletion mutations, replacing one sequence with another, adding a new sequence, manipulating RNAs
What would be the therapeutic strategy for anemia?
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
Gene editing in HSCs allows for ___________ expression in erythrocytes in adult patients
Gamma-globin (fetal globin)
What induces fetal globin expression in adult patients?
BCL11A knockout
What is expected from disrupting GATA-binding sequence in the BCL11A enhancer using CRISPR/Cas9?
Reduce BCL11A expression, reactivate gamma-globin expression
