A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

Autor: Haowen Zhou, Rita C.R. Perlingeiro, Nélio A. J. Oliveira, Carolina Ortiz-Cordero, Karim Azzag, Neha R. Dhoke, Qi Long Lu, James Kiley, Anne G. Bang, Sridhar Selvaraj, Hyunkee Kim, Sudheer Tungtur

Publikováno v: Cell Reports, Vol 36, Iss 2, Pp 109360-(2021)
Cell reports

SUMMARY Mutations in the fukutin-related protein (FKRP) gene result in a broad spectrum of muscular dystrophy (MD) phenotypes, including the severe Walker-Warburg syndrome (WWS). Here, we develop a gene-editing approach that replaces the entire mutan

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c05272cf41652c812199c3a48010a761
http://www.sciencedirect.com/science/article/pii/S2211124721007580

FUNGEMIA CAUSED BY Candida SPECIES IN A CHILDREN'S PUBLIC HOSPITAL IN THE CITY OF SÃO PAULO, BRAZIL: STUDY IN THE PERIOD 2007-2010

Autor: Nélio A. J. Oliveira, Claudete Rodrigues Paula, Luciana da Silva Ruiz, Débora Moreira, Rosane Christine Hahn, Angela Satie Nishikaku, Vanessa Kummer Perinazzo Oliveira, Analy Salles de Azevedo Melo

Publikováno v: Revista do Instituto de Medicina Tropical de São Paulo, Vol 56, Iss 4, Pp 301-305 (2014)
Revista do Instituto de Medicina Tropical de São Paulo, Volume: 56, Issue: 4, Pages: 301-305, Published: JUL 2014
Revista do Instituto de Medicina Tropical de São Paulo; Vol. 56 No. 4 (2014); 301-305
Revista do Instituto de Medicina Tropical de São Paulo; Vol. 56 Núm. 4 (2014); 301-305
Revista do Instituto de Medicina Tropical de São Paulo; v. 56 n. 4 (2014); 301-305
Revista do Instituto de Medicina Tropical de São Paulo
Instituto de Medicina Tropical (IMT)
instacron:IMT

Candidemia remains a major cause of morbidity and mortality in the health care environment. The epidemiology of Candida infection is changing, mainly in relation to the number of episodes caused by species C. non-albicans. The overall objective of th

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1a8f8f543583d2cb94da5714d07ac412
https://doi.org/10.1590/s0036-46652014000400006

Isolation of the pituitary gonadotrophic α-subunit hormone of the giant amazonian fish: pirarucu (Arapaima gigas)

Autor: João E. Oliveira, Paolo Bartolini, T. C. A. Sevilhano, P. R. E. Santo, Roberto Feitosa de Carvalho, Carlos R. J. Soares, Riviane Garcez, Marcos Tucunduva de Faria, Nélio A. J. Oliveira, C. F. P. Silva

Publikováno v: Fish Physiology and Biochemistry. 39:683-693

The cDNAs of the α-subunit of the pituitary gonadotrophic hormones (GTHα) of fish of the order Osteoglossiformes or the superorder Osteoglossomorpha have never been sequenced. For a better understanding the phylogenetic diversity and evolution of P

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f60df0b089e0702b943199d315c4a088
https://doi.org/10.1007/s10695-012-9730-1

Further evidence of association between mutations inFGFR2 and syndromic craniosynostosis with sacrococcygeal eversion

Autor: Maria Rita Passos-Bueno, Luis Garcia Alonso, Roberto D. Fanganiello, Nélio A. J. Oliveira

Publikováno v: Birth Defects Research Part A: Clinical and Molecular Teratology. 76:629-633

BACKGROUND: Pfeiffer syndrome (PS; OMIM #101600) is an autosomal dominant disorder characterized by craniosynostosis, midface hypoplasia, broad thumbs, brachydactyly, broad great toes, and variable syndactyly. CASE: We report a case of PS (type 3) wi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ecee361cb67f2bd5039889fcd5d99be9
https://doi.org/10.1002/bdra.20287

A novel homologous model for gene therapy of dwarfism by non-viral transfer of the mouse growth hormone gene into immunocompetent dwarf mice

Autor: Thomas G. Jensen, Alexander A. L. Jorge, Paolo Bartolini, Lars Aagaard, Eliza Higuti, Hanne Gissel, Frederick Dagnaes-Hansen, Cibele N. Peroni, Nélio A. J. Oliveira, Claudia R. Cecchi, Maria Jakobsen, Eliana R. Lima

Publikováno v: Cecchi, C R, Higuti, E, Oliveira, N A J, Lima, E R, Jakobsen, M, Dagnaes-Hansen, F, Gissel, H, Aagaard, L, Jensen, T G, Jorge, A A L, Bartolini, P & Peroni, C N 2014, ' A novel homologous model for gene therapy of dwarfism by non-viral transfer of the mouse growth hormone gene into immunocompetent dwarf mice ', Current Gene Therapy, vol. 14, no. 1, pp. 44-51 .

The possibilities for non-viral GH gene therapy are studied in immunocompetent dwarf mice (lit/lit). As expression vector we used a plasmid previously employed in immunodeficient dwarf mice (pUBI-hGH-gDNA) by replacing the human GH gene with the geno

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fecfaeaa718642e59b1e0a386908ab7c
https://pure.au.dk/portal/da/publications/a-novel-homologous-model-for-gene-therapy-of-dwarfism-by-nonviral-transfer-of-the-mouse-growth-hormone-gene-into-immunocompetent-dwarf-mice(f38f2fb6-0e77-4102-97ab-042e6738ccc9).html