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tissue engineering (architecture (summary table for the junctions…
tissue engineering
architecture
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cell types
mesenchymal: produce integrin (signalling), collagenase
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epithelial: feeder layer/coated with ECM, trypsin enzyme
squamous, cuboidal, columnar, pseudo stratified, transitional
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direct reprogramming
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examples
cardiomyocyte
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screening
epigenetic resetting
open circle: active, demethylated
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in vivo
myocardial infarction
retroviral transaction
express both alpha-actinin, and beta-Gal
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Gene therapy
gene correction
in vitro/ ex vivo
CF
mutation in CFTR, deletion of three codons, intestinal organoid will not swell under Forskolin treatment
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gene transfer
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virus
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non-integrating
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Aden-associated virus
ss-DNA, low immune response, small packaging capacity tho
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transfected packaging cell produce them, rather than inject virus directly
ex vivo/in vivo
LCA (in vivo), eye, low immune response, non-integrating AA virus, retinal cells don't divide tho, so information remain/virus injected in retina
ADA-SCID/BT (ex vivo), hematopoietic stem cell, retroviral transduction, refusion in bone marrow
PPK (ex vivo), dominate negative mutation, siRNA, stop the translation of the mRNA of mutated DNA
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natures gene therapy
revertant mosaicism: inherited disease-causing mutation is corrected by spontaneous genetic event in somatic cell
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RRR differences
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regenerate
liver
decellularized liver/hepatocyte/oxygen, growth factors, nutrition
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mutation
SNP
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non-synonymous
missense
EB (no production of collagen VII, separation of epidermis and dermal layer)
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nonsense (stop codon)
BT (mutation in several places, mutation in introns cause aberrant split, thus nonsense)
replace: cell: bone marrow, blood transfusion
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