CRISPR/Cas in iPSCs from sphingolipidoses patients

Moreira, Luciana; Duarte, Ana Joana; Ribeiro, Diogo; Amaral, Olga

http://hdl.handle.net/10400.18/6748

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Abstract(s)

Background: Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) were found as an immune adaptive mechanism in bacteria and quickly were applied to various fields as a promising tool for gene editing. Ultimately, the use of CRISPR/Cas-mediated gene editing can provide specific cellular models of disease, correct causal mutations in LSDs and create mutants for functional studies. Aim: In this work, experimental conditions were tested to validate a CRISPR/Cas9 approach to generate a KO. Conclusion: The cells were amenable to edition by CRISPR/Cas9 and the results obtained proved that editing was achieved. All results contribute to the improvement of our knowledge regarding this technology, broadening the validation of CRISPR application and making it an accessible option.

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This work was also financially supportedby National Funds through FCT—Fundacao da Ciencia e Tecno-logia (MCTES—Portugal) under Project ‘‘PTDC/BIM-MEC/4762/2014"