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The β-Globin Locus Control Region in Combination With the EF1α Short Promoter Allows Enhanced Lentiviral Vector-mediated Erythroid Gene Expression With Conserved Multilineage Activity

Autor: Aiste Monkeviciute, ME Alonso-Ferrero, Simone Scholz, Reiner Schulz, H. Bobby Gaspar, Marlene Carmo, Lin Zhang, Heba Saadeh, Manfred G. Schmidt, Yasuhiro Takeuchi, Michael Antoniou, Adrian J. Thrasher, Sean Knight, Lynette D. Fairbanks, Claudia Montiel-Equihua, Michael P. Blundell, Mary Collins

Publikováno v: Molecular Therapy; Vol 20

Some gene therapy strategies are compromised by the levels of gene expression required for therapeutic benefit, and also by the breadth of cell types that require correction. We designed a lentiviral vector system in which a transgene is under the tr

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Autor: PC Prada, José Luis Iribarren, Pilar Garrido, R MartinezSanz, JJ Jimenez, M Brouard, L. Perdomo, R Ávalos, J Montoto, R de la Llana, F. Benítez, ME Alonso

Publikováno v: Journal of Cardiothoracic Surgery

Results A cohort of 204 patients was studied. Age 51 +/9 years; 156 (76,5%) were male; Logistic Euroscore (LE) of 5.1 +/8.4, LVEF 58 +/11. Surgery was 86 (42.2%) CABG, 79 (38.7%) valvular, 16 (7.8%) combined surgery and 23 (11.3%) others. 28 (13.7%)

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6785970f0b7b866451ab6710ea1dcf44
https://doi.org/10.1186/1749-8090-10-s1-a311

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Bcr/Abl interferes with the Fanconi anemia/BRCA pathway: implications in the chromosomal instability of chronic myeloid leukemia cells

Autor: Ariana Jacome, Laura Fátima Asensi Pérez, Xabier Agirre, Helmut Hanenberg, Jordi Surrallés, María José Calasanz, Juan A. Bueren, ME Alonso-Ferrero, Paula Río, Maria Roser Pujol, Antonio Valeri, José A. Casado, Beatriz Albella, Felipe Prosper

Publikováno v: PLoS ONE, Vol 5, Iss 12, p e15525 (2010)
Recercat: Dipósit de la Recerca de Catalunya
Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Dadun. Depósito Académico Digital de la Universidad de Navarra
instname
PLoS ONE
Recercat. Dipósit de la Recerca de Catalunya
Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona

This work was supported by grants from the European Program "Life Sciences, Genomics and Biotechnology for Health" (CONSERT; Ref LSHB-CT-2004- 5242), Centro de Investigacio'n en Red de Enfermedades Raras (CIBERER), Comisión Interministerial de Cienc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9d308fe1c593c3bea64a2b2ca6bf6c66
http://europepmc.org/articles/PMC3011007?pdf=render

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Rescue of pyruvate kinase deficiency in mice by gene therapy using the human isoenzyme

Autor: José C. Segovia, Susana Navarro, ME Alonso-Ferrero, Maria Garcia-Gomez, José M. Bautista, Nestor W. Meza, Antonio Valeri, Juan A. Bueren, Oscar Quintana-Bustamante

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy. 17(12)

Human erythrocyte R-type pyruvate kinase deficiency (PKD) is a disorder caused by mutations in the PKLR gene that produces chronic nonspherocytic hemolytic anemia. Besides periodic blood transfusion and splenectomy, severe cases require bone marrow (

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ea742349d250b8938fd6fb2d923b90d
https://pubmed.ncbi.nlm.nih.gov/19755962

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Juvenile myoclonic epilepsy locus in chromosome 6p21.2-p11: linkage to convulsions and electroencephalography trait

Autor: Aw, Liu, Antonio V. Delgado-Escueta, Jm, Serratosa, Me, Alonso, Mt, Medina, Mn, Gee, Cordova S, Hz, Zhao, Jm, Spellman, Jr, Peek

Publikováno v: Europe PubMed Central

Despite affecting 4 million Americans and 100-200 million persons worldwide, the precise molecular mechanisms of human epilepsies remain unknown. Juvenile myoclonic epilepsy (JME) is the most frequent and, hence, most important form of hereditary gra

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::bdac7036da47ff0c9627724223c37738
https://pubmed.ncbi.nlm.nih.gov/7668263

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[Juvenile myoclonic epilepsy in chromosome 6p12: clinical and genetic advances]

Autor: Antonio V. Delgado-Escueta, Bai D, Bailey J, Mt, Medina, Me, Alonso, Morita R, Suzuki T, Ganesh S, Sugimoto T, Yamakawa K, Ochoa A, Jara-Prado A, Rasmussen A, Ramos-Peek M, Cordova S, Rubio-Donnadieu F

Publikováno v: Europe PubMed Central

Amongst idiopathic generalized epilepsies, juvenile myoclonic epilepsy (JME) is the most common, accounting for 12% to 30% of all epilepsies in the Western world. Classic JME consists of awakening myoclonias, grand mal convulsions and EEG 4 to 6 Hz p

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::3b5e418d3985bc8d268c6a631c1e430a
http://europepmc.org/abstract/med/12389199

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Mapping and positional cloning of common idiopathic generalized epilepsies: juvenile myoclonus epilepsy and childhood absence epilepsy

Autor: Antonio V. Delgado-Escueta, Mt, Medina, Jm, Serratosa, Ip, Castroviejo, Mn, Gee, Weissbecker K, Bw, Westling, Cy, Fong, Me, Alonso, Cordova S, Shah P, Khan S, Sainz J, Rubio-Donnadieu F, Rs, Sparkes

Publikováno v: Europe PubMed Central

Among the 40 to 100 million persons with epilepsy worldwide and the 2 to 2.5 million persons with epilepsies in the United States, approximately 50% have generalized epilepsies. Among all epilepsies, the most common are juvenile myoclonus epilepsy (J

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::f8ca5f34d5467479ffb7326cfe87de65
http://europepmc.org/abstract/med/10514826

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