Molecular Therapy Flashcards

1
Q

only totipotent cell

A

zygote

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2
Q

cells capable of forming all cells in the body except extraembryonic or placental cell

A

pluripotent cells: mesenchymal, endodermal, exodermal

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3
Q

cells that can differentiate into cells in a single cell lineage

A

multipotent (adult stem cells)

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4
Q

less potent cell lines with only few possible differentiation options available

A

oligopotent and unipotent

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5
Q

concerns about embryonic stem cells

A
  • ethical issues due to source
  • teratogenicity (accumulation of chromosomal mutations from multiple passages)
  • chromosomally unstable = malignant transformation
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6
Q

how are escs obtained

A

from blastocyst or inner cell mass during 1st week of gestation

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7
Q

creating esc via somatic nuclear transfer

A
  • remove nucleus of developing zygote and replace with adult donor cell
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8
Q

creating induced pluripotent stem cells

A
  • inducing transcription factors from earlier stage
  • uses protooncogenes
  • less risk for tumor formation
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9
Q

function of mesenchymal stromal cells

A
  • provide signals for growth and differentiation for resident stem cells (niches)
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10
Q

direct administration of mscs to target organ

A
  • smaller number of cells needed
  • dec immunogenicity
  • invasive
  • works via paracrine effect
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11
Q

systemic delivery homing of mscs

A
  • via iv infusion
  • provide immunomodulation
  • less invasive with similar efficacy
  • downside: pulmonary sequestration or in areas of inflammation (away from target organ)
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12
Q

stem cell therapy in cardiology

A
  • engraftment approaches

- gene modification of existing scar tissue

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13
Q

effect of msc on mi

A
  • decrease size of infarct
  • improve myocyte contractility and lvef in porcine models (high doses)
  • ecc shortening
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14
Q

stem cells in endo

A

hscs and mscs promote endogenous pancreatic islet cell regeneration (preserving or protecting islet cell)

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15
Q

types of cell based immunotherapy

A
  • immune modulation with mscs

- cancer immunotherapy

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16
Q

principle of dendritic cell therapy

A
  • isolate and grow patient dcs
  • expose them to tumor antigen lysate = prime to cancer
  • reintroduce dcs into patients
  • combats hiding tumor cells by directly exposing antigens to immune system
17
Q

example of dendritic cell therapy

A
  • sipuleucel t

- for castration-resistant prostate ca

18
Q

t/f cytokine induced killer cells require helper cell activation

A

false, they rely on activating and inhibiting receptors on targeted cells

19
Q

principles of cik therapy

A
  • expand and prime patient’s killer cells

- introduce with dct (more effective)

20
Q

what are adoptive t cell therapies

A
  • t cells that are genetically modified for specific effects

- car t cells

21
Q

fda approved anti cd19 car t cell therapies

A
  • tisgenlecleucel: b cell lymphoblastic leukemia

- axicabtagene ciloleucel: refractory large b cell lymphoma

22
Q

principles of gene therapy

A
  • gene transfer
  • gene silencing
  • gene/ome editing
23
Q

considerations for viral system in regenerative medicine

A
  • not harmful
  • can target specific populations of cells
  • can express long term if required
  • transgene expression regulatable
  • could target specific dna sequences within es cells
24
Q

use of mirnas

A
  • can target multiple rnas for degradation
  • can identify cancers
  • can be inhibited
25
Q

mirnas can inhibit ___ causing ___

A
  • inhibit d2 and d3 cyclin

- cell undergoes cell cycle arrest and activates cellular apoptosis pathways

26
Q

principle of genome editing

A
  • remove offending genetic component
  • crispr/cas9
  • only for single genes with high penetrance
27
Q

example of inducible gene expression

A
  • tet on/tet off inducible gene complexes (uses tetracycline)
28
Q

t/f in tissue engineering, autologous cells are preferred to avoid rejection

A

true

29
Q

cellular scaffold production using decellularization

A

read

30
Q

cellular scaffold production using adaptable biomaterials

A

read

31
Q

benefits of bioprinting

A
  • more complex structural arrangements of cell in 3d

- uses biogels and piezoelectric inkjet printing process

32
Q

t/f liposomes can cross the bb but are very sensitive and degrade quickly

A

true

33
Q

principle of gold nanoparticles

A
  • direct drugs to intended targets
  • minimize exposure to drug
  • lessen chemotherapeutic dose
34
Q

principle of iron oxide nanoparticles

A
  • magnetic and magnoelectric properties allow “payloads” to be directed to places they cannot reach