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As a prelude to translating these findings to the clinic, we developed and evaluated a novel 2-step clinically compliant procedure for manipulating peripheral blood stem cells (PBSC) to remove TN, preserve CD34+ hematopoietic stem cells, and provide for a fixed dose of memory T cells (TM) that includes T cells with specificity for common opportunistic pathogens encountered after HCT.
The described strategy to generate osteochondral plugs is simple to be implemented and – since it is based on clinically compliant cells and materials – is amenable to be readily tested in the clinic.
The derivation and culture of clinically compliant hPSC has been described[12] but limitations in scale of production and high costs remain significant challenges.
However, in our study we used 5% Stemulate (clinically compliant pooled human platelet lysate) to provide growth factors and other proteins to support proliferation of the sorted cells.
The study will take advantage of a clinically compliant protocol involving the establishment of autologous hiPSCs from each of the study subjects and a differentiation method to generate retinal pigment epithelium cells for subsequent transplantation back to the patients.
Here we show that multiple cellular products currently being considered for therapy can be generated from a single master cell bank of this or any other clinically compliant iPSC line.
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Yet there remain cases that were clinically compatible with 4s stage but not fully compliant with the expected marker profile [ 9- 12].
Only 4.3% of cases where quinolone prescriptions were not formulary guideline compliant were felt to be appropriate clinically (as defined above), with no difference before or after OSA implementation.
We successfully transduced Fabry patient CD34+ hematopoietic cells with "near-clinical grade" LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility.
We optimized the co-culture ratio of K562 aAPCs to immune cells, and migrated this method to a G-Rex cell growth platform to derive clinically relevant cell numbers in a Good Manufacturing Practice (GMP -compliant manner.
Compliant patients who had a clinical response of either cure or failure at TOC were considered clinically evaluable (CE).
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