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Cell therapy for haemophilia is based, mainly, in transplantation of healthy cells to replace the deficient function, for example, the transplantation of liver sinusoidal endothelial cells or endothelial progenitor cells derived from iPSCs.
4 The treatment aim in CAH is to replace the deficient hormones (glucocorticoid ± mineralocorticoid) and reduce the excessive androgens.
The primary goal of treating classic CAH is to reduce the excess adrenal androgen production and replace the deficient hormones, namely cortisol and aldosterone.
This translational investigation of a potential oral therapy for a genetic-based mitochondrial RC disorder revealed that endogenous upregulation of CoQ content by probucol treatment is far more effective than attempts to replace the deficient metabolite (CoQ) by exogenous CoQ10 supplementation.
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Nucleoside imbalance is thought to underlie disease pathogenesis in the latter group, and in theory replacing the deficient deoxyribonucleosides might correct these disorders.
Where cartilage production is deficient, fibroblasts replace the region with generalized fibrous tissue.
Hence, there will be a need to replenish the deficient cells and associated function and/or to replace the diseased tissue or organ.
The bridge is structurally deficient and the cost to replace the bridge would amount to $591,000.
In RP-deficient zebrafish, we found activation of both nucleotide catabolism and biosynthesis, which is consistent with the need to break and replace the faulty ribosomal RNA.
Then replace the wick.
Replace the air conditioner.
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