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Akademický článek

The impact of COVID-19 on rare metabolic patients and healthcare providers: results from two MetabERN surveys

Autor: C. Lampe, C. Dionisi-Vici, C. M. Bellettato, L. Paneghetti, C. van Lingen, S. Bond, C. Brown, A. Finglas, R. Francisco, S. Sestini, J. M. Heard, M. Scarpa, MetabERN collaboration group

Publikováno v: Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-14 (2020)

Abstract The ongoing coronavirus disease 2019 (COVID-19) pandemic has caused disruption in all aspects of daily life, including the management and treatment of rare inherited metabolic disorders (IMDs). To perform a preliminary assessment of the inci

Externí odkaz: https://doaj.org/article/f3096edf4ab6405ab6b5b184ee678d23

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Additional file 2 of The impact of COVID-19 on rare metabolic patients and healthcare providers: results from two MetabERN surveys

Autor: C. Lampe, C. Dionisi-Vici, C. M. Bellettato, L. Paneghetti, C. Van Lingen, S. Bond, C. Brown, A. Finglas, R. Francisco, S. Sestini, J. M. Heard, Scarpa, M.

Additional file 2. Original survey sent to PO. List of questions and possible answers included in the PO survey.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c60a7fb0c831a8657f758a68d46ec40e

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Additional file 1 of The impact of COVID-19 on rare metabolic patients and healthcare providers: results from two MetabERN surveys

Autor: C. Lampe, C. Dionisi-Vici, C. M. Bellettato, L. Paneghetti, C. Van Lingen, S. Bond, C. Brown, A. Finglas, R. Francisco, S. Sestini, J. M. Heard, Scarpa, M.

Additional file 1. Original survey sent to HCPs. List of questions and possible answers included in the HCPs survey.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c27b66c01a3459ec27958758254e7c1e

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In situ retrovirus-mediated gene transfer into the liver

Autor: N, Ferry, S, Branchereau, J M, Heard, O, Danos

Publikováno v: Methods in molecular medicine. 7

Gene transfer into hepatocytes is a promising approach for the treatment of genetic liver diseases. Candidate diseases for human trials are life-threatening disorders resulting from a single genetic defect that do not compromise other liver functions

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::f58463986892859b48f57d2c1fc07ee8
https://pubmed.ncbi.nlm.nih.gov/24493427

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[Untitled]

Autor: J.-M. Heard, D. Bohl

Publikováno v: Cell Biology and Toxicology. 14:83-94

Many diseases that are candidates for gene therapy require that the therapeutic gene expression level be controlled to ensure biological efficacy and to prevent toxic effects. Various systems have been described that allow transcriptional regulation

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::05142bb5d7ab4e344d0971b966bf249f
https://doi.org/10.1023/a:1007480419685

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A cell surface marker gene transferred with a retroviral vector into CD34+ cord blood cells is expressed by their T-cell progeny in the SCID- hu thymus

Autor: E. Vilmer, G Dranoff, Bruno Péault, V Marechal, I Khazaal, P Blot, S Fournier, O Danos, J M Heard, N Schlegel, O Schwartz, C Champseix, P Lehn

Publikováno v: Blood. 88:107-113

Gene transduction into immature hematopoietic cells collected at birth from the umbilical cord could be useful for the treatment of genetic or acquired disorders of the hematopoietic system diagnosed during pregnancy. The SCID-hu mouse is a convenien

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::84f517531c5457b8f51f6ac08808a3bb
https://doi.org/10.1182/blood.v88.1.107.107

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Tetracycline-Controlled Transactivators and Their Potential Use in Gene Therapy Applications

Autor: D. Bohl, J.-M. Heard

Publikováno v: Transcription Factors ISBN: 9783642623615

The control of gene expression in higher eukaryotes has developed into an invaluable tool for both the design of gene therapy strategies and the study of gene function. Application of gene therapy to a number of diseases will require that therapeutic

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::8b15d8053e397fc6776760c5f72e1f5c
https://doi.org/10.1007/978-3-642-18932-6_17

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Akademický článek

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Delivering erythropoietin through genetically engineered cells

Autor: D, Bohl, J M, Heard

Publikováno v: Journal of the American Society of Nephrology : JASN. 11

Erythropoietin (Epo) is a glycoprotein hormone produced by genetic engineering. Many pathologic conditions could benefit from its administration, such as chronic renal failure or hemoglobinopathies. Epo secretion from genetically modified tissued cou

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::61edee8b9b886e830aefd1890b122ead
https://pubmed.ncbi.nlm.nih.gov/11065350

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Improvement of erythropoiesis in beta-thalassemic mice by continuous erythropoietin delivery from muscle

Autor: D, Bohl, A, Bosch, A, Cardona, A, Salvetti, J M, Heard

Publikováno v: Blood. 95(9)

beta-Thalassemias are highly prevalent genetic disorders that can cause severe hemolytic anemia. The main pathophysiologic feature of beta-thalassemia is the accumulation of unpaired alpha-globin chains in erythrocyte precursors and red blood cells (

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::22907180c00d84b753e25356538e2bee
https://pubmed.ncbi.nlm.nih.gov/10779423

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