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Gene edition by CRISPR-Cas9 in the correction of Duchenne muscular dystrophy in a canine model (GRMD) from induced pluripotent stem cells

Grant number: 15/09575-1
Support type:Scholarships in Brazil - Post-Doctorate
Effective date (Start): August 01, 2015
Effective date (End): February 28, 2017
Field of knowledge:Agronomical Sciences - Veterinary Medicine
Cooperation agreement: Coordination of Improvement of Higher Education Personnel (CAPES)
Principal Investigator:Carlos Eduardo Ambrósio
Grantee:Natalia Juliana Nardelli Gonçalves
Home Institution: Faculdade de Zootecnia e Engenharia de Alimentos (FZEA). Universidade de São Paulo (USP). Pirassununga , SP, Brazil
Associated research grant:13/08135-2 - CTC - Center for Cell-Based Therapy, AP.CEPID


The induced reprogramming strategies are being employed in order to induce somatic cells to a pluripotent state, similar to the embryonic. The nuclear reprogramming process is highly desirable and has significant contributions from the study of basic and applied science, for example, increasing the efficiency of animal production of biotechnical and medicine, with the possibility of autologous cell therapy for the treatment of many diseases. The Duchenne Muscular Dystrophy (DMD) is a genetic disease that causes progressive degeneration of the skeletal muscles. The gene encoding dystrophin protein is composed of 79 exons, and its size makes it very susceptible to mutations. The Golden Retriever Muscular Dystrophy (GRMD) is genetically homologous to DMD that affects humans, and this animal model is what most resembles the mutation in humans, with a point mutation, where the exon 7 is deleted, causing a premature stop in exon 8. Therefore, the aim of this study is to produce IPS from dog fibroblasts affected by Duchenne Muscular Dystrophy, characterize these cells and submit them to CRISPR-Cas9 gene edition, to correct the site mutation, generating healthy cells as a model for future studies in animals and humans. The genetic correction of GRMD into iPS cells expands a newly explored field, allowing the correction in the Golden Retriever model, screening this gene and mutation site. Basing future research in humans, enabling the correction of a degenerative genetic disease, and the development of a new model. (AU)

Scientific publications
(References retrieved automatically from Web of Science and SciELO through information on FAPESP grants and their corresponding numbers as mentioned in the publications by the authors)
GONCALVES, N. J. N.; BRESSAN, F. F.; ROBALLO, K. C. S.; MEIRELLES, F. V.; XAVIER, P. L. P.; FUKUMASU, H.; WILLIAMS, C.; BREEN, M.; KOH, S.; SPER, R.; PIEDRAHITA, J.; AMBROSIO, C. E. Generation of LIF-independent induced pluripotent stem cells from canine fetal fibroblasts. Theriogenology, v. 92, p. 75-82, APR 1 2017. Web of Science Citations: 10.

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