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(Reference retrieved automatically from Web of Science through information on FAPESP grant and its corresponding number as mentioned in the publication by the authors.)

A fast method to reprogram and CRISPR/Cas9 gene editing from erythroblasts

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Melo, Uira Souto [1] ; Leite, Felipe de Souza [1] ; Costa, Silvia [1] ; Rosenberg, Carla [1] ; Zatz, Mayana [1]
Total Authors: 5
[1] Univ Sao Paulo, Human Genome & Stem Cell Res Ctr, Biosci Inst, Dept Genet & Evolutionary Biol, BR-05508900 Sao Paulo, SP - Brazil
Total Affiliations: 1
Document type: Journal article
Source: STEM CELL RESEARCH; v. 31, p. 52-54, AUG 2018.
Web of Science Citations: 1

An efficient one-step procedure to reprogram fibroblasts into human induced pluripotent stem cells (hiPSC) and perform CRISPR/Cas9 gene editing simultaneously was recently reported. Here we show that such simultaneous reprogramming and gene editing can be efficiently done from erythroblasts. We successfully obtained human induced pluripotent stem cells colonies together with in frame and out of frame CAPN1 mutations in one or both alleles. We did not identify off-targets in edited cell lines. The entire process, from blood collection to mutated hiPSC took approximately 5 weeks, a much shorter period than standard multi-step methodologies using fibroblasts. Noteworthy, blood drawing is a less invasive procedure than a skin biopsy. (AU)

FAPESP's process: 16/22318-0 - Alternative approaches targeting Duchenne Muscular Dystrophy treatment
Grantee:Felipe de Souza Leite
Support type: Scholarships in Brazil - Post-Doctorate
FAPESP's process: 16/14517-3 - Search for "protective" modifying variants/mechanisms in asymptomatic or very mildly affected individuals with pathogenic mutation in SPAST gene
Grantee:Uirá Souto Melo
Support type: Scholarships in Brazil - Post-Doctorate
FAPESP's process: 13/08028-1 - CEGH-CEL - Human Genome and Stem Cell Research Center
Grantee:Mayana Zatz
Support type: Research Grants - Research, Innovation and Dissemination Centers - RIDC