Río, Paula Navarro, Susana Guenechea, Guillermo Sánchez-Domínguez, Rebeca Lamana, Maria Luisa Yañez, Rosa Casado, Jose A Mehta, Parinda A Pujol, Maria Roser Surrallés, Jordi
...
Published in
Blood
Previous Fanconi anemia (FA) gene therapy studies have failed to demonstrate engraftment of gene-corrected hematopoietic stem and progenitor cells (HSPCs) from FA patients, either after autologous transplantation or infusion into immunodeficient mice. In this study, we demonstrate that a validated short transduction protocol of G-CSF plus plerixafo...
Flotte, Terence R.
Within American biomedical research, as supported primarily by the National Institutes of Health (NIH) since the post–World War II era, it has been considered axiomatic that investments in discovery research will ultimately lead to the development of novel approaches to diagnostics and therapeutics that will improve the health and well-being of cit...
Moiani, Arianna Miccio, Annarita Rizzi, Ermanno Severgnini, Marco Pellin, Danilo Suerth, Julia Debora Baum, Christopher De Bellis, Gianluca Mavilio, Fulvio
Published in
PLoS One
Barde, I Laurenti, E Verp, S Wiznerowicz, M Offner, S Viornery, A Galy, A Trumpp, A Trono, D
Published in
Gene therapy
Insertional mutagenesis represents a serious adverse effect of gene therapy with integrating vectors. However, although uncontrolled activation of growth-promoting genes in stem cells can predictably lead to oncological processes, this is far less likely if vector transcriptional activity can be restricted to fully differentiated cells. Diseases re...
Ni, W Le Guiner, C Gernoux, G Penaud-Budloo, M Moullier, P Snyder, R O
Published in
Gene therapy
Legitimate uses of gene transfer technology can benefit from sensitive detection methods to determine vector biodistribution in pre-clinical studies and in human clinical trials, and similar methods can detect illegitimate gene transfer to provide sports-governing bodies with the ability to maintain fairness. Real-time PCR assays were developed to ...
Charrier, S Ferrand, M Zerbato, M Précigout, G Viornery, A Bucher-Laurent, S Benkhelifa-Ziyyat, S Merten, O Perea, J Galy, A
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International audience
Flotte, T R
Published in
Gene therapy
Lock, Martin McGorray, Susan Auricchio, Alberto Ayuso, Eduard Beecham, E. Jeffrey Blouin-Tavel, Veronique Bosch, Fatima Bose, Mahuya Bryne, Barry J. Caton, Tina
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A recombinant adeno-associated virus serotype 2 Reference Standard Material (rAAV2 RSM) has been produced and characterized with the purpose of providing a reference standard for particle titer, vector genome titer, and infectious titer for AAV2 gene transfer vectors. Production and purification of the reference material were carried out by helper ...
Mueller, Christian Strayer, M. S. Sirninger, Jeffrey Braag, Sofia A. Branco, Francisco Louboutin, Jean-Pierre Flotte, Terence R. Strayer, David S.
In cystic fibrosis (CF), respiratory failure caused by progressive airway obstruction and tissue damage is primarily a result of the aberrant inflammatory responses to lung infections with Pseudomonas aeruginosa. Despite considerable improvement in patient survival, conventional therapies are mainly supportive. Recent progress toward gene therapy f...
Beattie, Stuart G Goetzman, Eric Tang, Qiuishi Conlon, Thomas Campbell-Thompson, Martha Matern, Dietrich Vockley, Jerry Flotte, Terence R
Published in
The journal of gene medicine
Very long chain acyl coenzyme A (CoA) dehydrogenase (VLCAD) deficiency is a relatively common mitochondrial beta-oxidation disorder. The most severe form of VLCAD deficiency presents with neonatal cardiomyopathy and hepatic failure and is generally fatal within the first year of life. Mice deficient for long chain acyl CoA dehydrogenase (LCAD) clos...
