Results 111 to 120 of about 172,174 (293)
Studies of the adenovirus 5 L1 gene aimed at developing L1 gene deficiencies for use in gene therapy vectors [PDF]
Gene therapy is a novel approach to the treatment of human disease that is in its very early stages of development. Its purpose is to add to and/or alter the pattern of gene expression in cells so as to achieve a therapeutic benefit and is being ...
Arslanoglu, Alper
core
Alpha-1 antitrypsin deficiency (AATD) is characterized by both chronic lung disease due to loss of wild-type AAT (M-AAT) antiprotease function and liver disease due to toxicity from delayed secretion, polymerization, and aggregation of misfolded mutant ...
Kondratov, Oleksandr +11 more
core +1 more source
Molecular Oncology, EarlyView.Drug resistance limits treatment success in a subset of lung cancers driven by ROS1 gene alterations. Using patient‐derived cells and computer simulations, we studied three key mutations and how they affect five targeted drugs. The mutations reduced drug effectiveness in different ways by altering protein structure and behavior.
Farhan Ul Haq +8 more
wiley +1 more source
ApoE gene therapy to treat hyperlipidemia and atherosclerosis
, 2006 Atherosclerosis is the leading cause of death in industrialized countries and is becoming an increasingly worldwide risk to health. Apolipoprotein E (ApoE) is a blood circulating protein with pleiotropic atheroprotective properties that has emerged as a ...
Owen, JS +3 more
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Review of Treatment for Adenosine Deaminase Deficiency (ADA) Severe Combined Immunodeficiency (SCID)
Therapeutics and Clinical Risk Management, 2022 Elizabeth Secord,1 Nicholas L Hartog2 1Pediatrics, Division of Allergy and Immunology, Wayne State University School of Medicine, Detroit, MI, USA; 2Pediatrics, Division of Allergy and Immunology, Michigan State University College of Human Medicine ...
Secord E, Hartog NL
doaj
Molecular Oncology, EarlyView.BCL9 and BCL9L drive bladder cancer progression by enhancing β‐catenin signaling, promoting proliferation, migration, invasion, and organoid growth. Genetic depletion of BCL9(L) suppresses malignant phenotypes, while pharmacological disruption of the β‐catenin/BCL9(L) complex with ZW4864 inhibits canonical Wnt signaling and tumor‐associated cellular ...
Roland Kotolloshi +11 more
wiley +1 more source
Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology [PDF]
, 2013 Spinal muscular atrophy (SMA) is a genetic disease which is characterized by muscle weakness and atrophy. The disease arises from mutations in the survival motor neuron 1 (SMN1) gene causing degeneration of spinal cord motor neurons.
Little, Daniel
core
FEBS Open Bio, EarlyView.ERα splice variant ERα∆7 lacks the C‐terminus, and its expression may change phenotypes of breast cancers. Our results showed that ERα∆7 is found in the luminal A subtype, and elevated ERα∆7 levels are linked to improved cell survival with lower proliferation and migration.
Long Wai Tsui +10 more
wiley +1 more source
Neuroprotective gene therapy for Parkinson's disease.
, 2002 Parkinson's disease (PD) is a neurodegenerative disease characterised by a progressive loss of the dopaminergic neurones in the substantia nigra pars compacta.
Lehtonen, E. +7 more
core
KCNQ1 suppression-replacement gene therapy in transgenic rabbits with type 1 long QT syndrome. [PDF]
BACKGROUND AND AIMS Type 1 long QT syndrome (LQT1) is caused by pathogenic variants in the KCNQ1-encoded Kv7.1 potassium channels, which pathologically prolong ventricular action potential duration (APD). Herein, the pathologic phenotype in transgenic
Alerni, Nicolo +31 more
core +1 more source