Results 101 to 110 of about 11,459,182 (339)

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

TOMM20 as a driver of cancer aggressiveness via oxidative phosphorylation, maintenance of a reduced state, and resistance to apoptosis

Molecular Oncology, EarlyView.
TOMM20 increases cancer aggressiveness by maintaining a reduced state with increased NADH and NADPH levels, oxidative phosphorylation (OXPHOS), and apoptosis resistance while reducing reactive oxygen species (ROS) levels. Conversely, CRISPR‐Cas9 knockdown of TOMM20 alters these cancer‐aggressive traits.
Ranakul Islam, Megan E. Roche, Zhao Lin, Diana Whitaker‐Menezes, Victor Diaz‐Barros, Eurico Serrano, Maria Paula Martinez Cantarin, Nancy J. Philp, Atrayee Basu Mallick, Ubaldo Martinez‐Outschoorn   +9 more
wiley   +1 more source

Gene therapy for epilepsy [PDF]

Epilepsy & Behavior, 2014
Gene therapy may represent an effective alternative to standard pharmacological approaches for certain forms of epilepsy. Currently, the best candidates for this therapeutic approach appear to be epilepsies characterized by a focal lesion. Gene therapy has been attempted to produce antiepileptogenic (prevention of development of epilepsy in subject at ...
openaire   +5 more sources

AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo

Journal of Extracellular Vesicles
MPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available.
Tierra A. Bobo, Michael Robinson, Christopher Tofade, Marina Sokolski‐Papkov, Peter Nichols, Stephen Vorobiov, Haiyan Fu   +6 more
doaj   +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

Viral Vectors in Gene Therapy

Diseases, 2018
Applications of viral vectors have found an encouraging new beginning in gene therapy in recent years. Significant improvements in vector engineering, delivery, and safety have placed viral vector-based therapy at the forefront of modern medicine.
K. Lundstrom
semanticscholar   +1 more source

Plasma lipidomic and metabolomic profiles in high‐grade glioma patients before and after 72‐h presurgery water‐only fasting

Molecular Oncology, EarlyView.
Presurgery 72‐h fasting in GB patients leads to adaptations of plasma lipids and polar metabolites. Fasting reduces lysophosphatidylcholines and increases free fatty acids, shifts triglycerides toward long‐chain TGs and increases branched‐chain amino acids, alpha aminobutyric acid, and uric acid.
Iris Divé, Lisa Hahnefeld, Katharina J. Wenger, Donat Kögel, Joachim Steinbach, Gerd Geisslinger, Michael W. Ronellenfitsch, Irmgard Tegeder   +7 more
wiley   +1 more source

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

, 1983
Henry I. Miller
openalex   +1 more source

Tumor angiogenesis and anti-angiogenic gene therapy for cancer

Oncology Letters, 2018
When Folkman first suggested a theory about the association between angiogenesis and tumor growth in 1971, the hypothesis of targeting angiogenesis to treat cancer was formed.
Ting-Chuan Li, G. Kang, T. Wang, He Huang   +3 more
semanticscholar   +1 more source