Results 21 to 30 of about 1,008,568 (216)

Tailoring therapy for heart failure: the pharmacogenomics of adrenergic receptor signaling [PDF]

, 2014
Grazia Daniela Femminella,1 Vincenzo Barrese,2,3 Nicola Ferrara,1,4 Giuseppe Rengo4 1Department of Translational Medical Sciences, Federico II University, Naples, Italy; 2Department of Neuroscience, Reproductive Science and Odontostomatology, Federico II
Ferrara, N, Barrese V, Ferrara N, FERRARA, NICOLA, Rengo, G, Barrese, Vincenzo, FEMMINELLA, GRAZIA DANIELA, Femminella GD, Barrese, V, Rengo G, Femminella, GD, RENGO, GIUSEPPE   +11 more
core   +1 more source

Translating the combination of gene therapy and tissue engineering for treating recessive dystrophic epidermolysis bullosa [PDF]

European Cells & Materials, 2018
The combination of gene therapy and tissue engineering is one of the most promising strategies for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
A Dakiw Piaceski, D Larouche, K Ghani, F Bisson, S Cortez Ghio, S Larochelle, VJ Moulin, M Caruso, L Germain   +8 more
doaj   +1 more source

Genome-wide association study of genetic determinants of LDL-c response to atorvastatin therapy: importnace of Lp(a) [PDF]

, 2012
We carried out a genome-wide association study (GWAS) of LDL-c response to statin using data from participants in the Collaborative Atorvastatin Diabetes Study (CARDS; n = 1,156), the Anglo-Scandinavian Cardiac Outcomes Trial (ASCOT; n = 895), and the ...
Postmus, Iris, Colhoun, H.M., McKeigue, Paul M, Stanton, A.V., Trompet, S., Hyde, Craig, Colhoun, Helen M, Caulfield, M., Shields, Denis C., Hitman, Graham A, CARDS, Poulter, Neil, Shields, D.C., Sever, P., Chatterjee, Aurobindo, Trompet, Stella, Durrington, Paul N, Deshmukh, H.A., DeMicco, D.A., Calle, Roberto A., Hitman, Graham A., Neil, A., McKeigue, Paul M., Fuller, John H., Postmus, I., Hitman, G.A., DeMicco, David A., Johnson, T., ?, ?, Calle, Roberto A, Betteridge, D.J., Livingstone, Shona, Ford, Ian, McKeigue, P.M., Stanton, Alice V., Jukema, J.W., Johnson, Toby, Durrington, Paul N., Neil, Andrew, PROSPER Investigators, Livingstone, S., Jukema, J. Wouter, ASCOT, Colhoun, Helen, Ford, I., Sever, Peter, Fuller, J.H., Jukema, J Wouter, Caulfield, Mark, Fuller, John H, Chatterjee, A., Deshmukh, Harshal A, Calle, R.A., Hyde, C., Charlton-Menys, V., Poulter, N., Charlton-Menys, Valentine, DeMicco, David A, Betteridge, D John, McKeigue, Paul M.; id_orcid, Betteridge, D. John, Durrington, P.N., Colhoun, Helen M., Shields, Denis C, Deshmukh, Harshal A., Stanton, Alice V   +65 more
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Beta-Ketothiolase Deficiency: A Comprehensive Review of Genetic Variants and Pathophysiology [PDF]

Annals of Child Neurology
Beta-ketothiolase deficiency (BKD) is a rare autosomal recessive disorder caused by mutations in the ACAT1 gene, also known as mitochondrial acetoacetyl-coenzyme A thiolase (MAT) deficiency.
Sohit Kashyap, Anil Kumar, Anita Choudhary, Ajay Kumar, Arvinder Wander, Anjana Munshi   +5 more
doaj   +1 more source

Genetic therapy for the nervous system [PDF]

Human Molecular Genetics, 2011
Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising.
William J, Bowers, Xandra O, Breakefield, Miguel, Sena-Esteves   +2 more
openaire   +2 more sources

Pharmacogenetics of Migraine: genetic variants and their potential role in migraine therapy [PDF]

, 2007
Migraine is a paroxysmal neurological disorder affecting up to 6% of males and 18% of females in the general population, and has been demonstrated to have a strong, but complex, genetic component.
LR Griffiths, Fernandez, F, Fernandez, Francesca, Griffiths, Lyn R., Griffiths, LR, Colson, Natalie, Colson, N. J., Griffiths, Lyn, NJ Colson, F Fernandez, Colson, NJ   +10 more
core   +1 more source

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice [PDF]

, 2022
α1-antitrypsin deficiency is a rare genetic condition that can cause liver and/or lung disease. There is currently no cure for this disorder, although repeated infusions of plasma-purified protein may slow down emphysema progression.
Zieger, Marina, Greer, Cynthia, Flotte, Terence R, Mueller, Christian, Borel, Florie, Blackwood, Meghan, Gernoux, Gwladys   +6 more
core   +2 more sources

Cellular and genetic therapies for haemophilia [PDF]

Haemophilia, 2006
Summary.  Haemophilia continues to be a prime target for a variety of gene and cell‐based therapies. Pre‐clinical successes in both mouse and dog models of the disease have been documented with a variety of approaches over the past decade, and there have now been six small clinical trials of gene transfer in haemophilia.
Lillicrap, David, VandenDriessche, T., High, K.   +2 more
openaire   +3 more sources

Association of IFNL3 rs12979860 and rs8099917 with biochemical predictors of interferon responsiveness in chronic hepatitis C virus infection [PDF]

, 2013
Background & Aims: Genetic variations near the interferon lambda 3 gene (IFNL3, IL28B) are the most powerful predictors for sustained virologic response (SVR) in patients with chronic hepatitis C virus (HCV) infection, compared to other biochemical or ...
Simone Susser (48294), Annika Brodzinski, Thomas Berg (186372), Migaud, Pascal, Jacob George (205614), Brodzinski, Annika, Tobias Müller, Susser, Simone, Stephan Böhm (334611), Sarrazin, Christoph, Christoph Sarrazin (48298), Pascal Migaud, Schott, Eckart, Bömmel, Florian van, Heiko Witt, Florian van Bömmel, Janett Fischer, Christoph Sarrazin, Stewart, Graeme, Graeme Stewart, Witt, Heiko, Thomas Berg, Simone Susser, Balazs Fülöp, Fülöp, Balazs, Stephan Böhm, Annika Brodzinski (477753), Balazs Fülöp (477754), Müller, Tobias, Fischer, Janett, Böhm, Stephan, Berg, Thomas, Tobias Müller (163591), Janett Fischer (477751), Eckart Schott (320945), Graeme Stewart (208243), Jacob George, Heiko Witt (161481), Pascal Migaud (477752), George, Jacob, Eckart Schott, Florian van Bömmel (477755)   +41 more
core   +1 more source

Genetic therapies against HIV [PDF]

Nature Biotechnology, 2007
Highly active antiretroviral therapy prolongs the life of HIV-infected individuals, but it requires lifelong treatment and results in cumulative toxicities and viral-escape mutants. Gene therapy offers the promise of preventing progressive HIV infection by sustained interference with viral replication in the absence of chronic chemotherapy.
John J, Rossi, Carl H, June, Donald B, Kohn   +2 more
openaire   +2 more sources