Results 151 to 160 of about 12,497,887 (407)

Gene Therapy Tools for Brain Diseases

Frontiers in Pharmacology, 2019
Neurological disorders affecting the central nervous system (CNS) are still incompletely understood. Many of these disorders lack a cure and are seeking more specific and effective treatments.
S. Ingusci, G. Verlengia, M. Soukupova, S. Zucchini, M. Simonato   +4 more
semanticscholar   +1 more source

Solid-phase-assisted synthesis of targeting peptide-PEG-oligo(ethane amino)amides for receptor-mediated gene delivery. [PDF]

, 2012
In the forthcoming era of cancer gene therapy, efforts will be devoted to the development of new efficient and non-toxic gene delivery vectors. In this regard, the use of Fmoc/Boc-protected oligo(ethane amino)acids as building blocks for solid-phase ...
Akinc, Aumailley, Behr, Blessing, Boussif, Breunig, Carlos Mas-Moruno, Chollet, Christian Dohmen, Christina Troiber, Cotten, Cox, David Schaffert, Davis, Davis, Dechantsreiter, Desgrosellier, Drake, Edinger, Ellman, Erbacher, Ernest Giralt, Ernst Wagner, Felding-Habermann, Felgner, Fella, Grandinetti, Harbottle, Hartmann, Hartmann, Haubner, Horst Kessler, Irene Martin, J., Kantlehner, Kichler, Kim, Kursa, Li, Lin, Luthman, Madder, Mas-Moruno, McKenzie, Meritxell Teixidó, Merkel, Meyer, Meyer, Michael Günther, Moghimi, Mosca, Nie, Oba, Oupicky, Pack, Petra Kos, Pfaff, Plank, Reis, Ruoslahti, Russ, Russ, Schaffert, Schaffert, Schaffert, Schiffelers, Sonawane, Trubetskoy, Ulrich Lächelt, Varkouhi, Wagner, Wagner, Wagner, Wagner, Walker, Wu, Xia, Xu, Zintchenko   +78 more
core   +3 more sources

Expression and DNA methylation of 20S proteasome subunits as prognostic and resistance markers in cancer

Molecular Oncology, EarlyView.
Comprehensive analysis of genomic mutations, gene expression, DNA methylation, and pathway analysis of TCGA data was carried out to define cancer types in which proteasome subunits expression is associated with worse survival. Albeit the effect of specific proteasome subunits on cellular function, the main role of the proteasome is better evaluated ...
Ruba Al‐Abdulla, Simone Venz, Ruslan Al‐Ali, Martin Wendlandt, Mandy Radefeldt, Elke Krüger   +5 more
wiley   +1 more source

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC

Disease Models & Mechanisms, 2016
Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe lysosomal storage disease caused by deficiency in activity of the transmembrane enzyme heparan-α-glucosaminide N-acetyltransferase (HGSNAT) that catalyses the N-acetylation of α-glucosamine residues ...
Sara Marcó, Anna Pujol, Carles Roca, Sandra Motas, Albert Ribera, Miguel Garcia, Maria Molas, Pilar Villacampa, Cristian S. Melia, Víctor Sánchez, Xavier Sánchez, Joan Bertolin, Jesús Ruberte, Virginia Haurigot, Fatima Bosch   +14 more
doaj   +1 more source

Gene Therapy for Human Genetic Disease? [PDF]

, 1972
Elizabeth F. Neufeld, Charles C. Sweeley
openalex   +1 more source

Gene Therapy in Cardiac Arrhythmias [PDF]

, 2006
Gene therapy has progressed from a dream to a bedside reality in quite a few human diseases. From its first application in adenosine deaminase deficiency, through the years, its application has evolved to vascular angiogenesis and cardiac arrhythmias.
Francis, Johnson, Praveen, S.V, Venugopal, K   +2 more
core  

Detecting homologous recombination deficiency for breast cancer through integrative analysis of genomic data

Molecular Oncology, EarlyView.
This study develops a semi‐supervised classifier integrating multi‐genomic data (1404 training/5893 validation samples) to improve homologous recombination deficiency (HRD) detection in breast cancer. Our method demonstrates prognostic value and predicts chemotherapy/PARP inhibitor sensitivity in HRD+ tumours.
Rong Zhu, Katherine Eason, Suet‐Feung Chin, Paul A. W. Edwards, Raquel Manzano Garcia, Richard Moulange, Jia Wern Pan, Soo Hwang Teo, Sach Mukherjee, Maurizio Callari, Carlos Caldas, Stephen‐John Sammut, Oscar M. Rueda   +12 more
wiley   +1 more source

Gene Therapy: Not to be Feared or Over–Regulated [PDF]

, 1983
Henry I. Miller
openalex   +1 more source

AAV vectors: The Rubik’s cube of human gene therapy

Molecular Therapy, 2022
A. Pupo, Audry Fernández, Siew Hui Low, Achille François, Lester Suárez-Amarán, R. Samulski   +5 more
semanticscholar   +1 more source

The C9ORF72 mutation brings more answers and more questions. [PDF]

, 2013
The clinical, neuropsychiatric and neuroimaging features of patients who carry the important new C9ORF72 mutation are discussed in this special series of Alzheimer's Research & Therapy.
Miller, Bruce L
core   +1 more source