Metabolomic Profiling of Statin Use and Genetic Inhibition of HMG-CoA Reductase [PDF]
, 2015 Background Statins are first-line therapy for cardiovascular disease prevention, but their systemic effects across lipoprotein subclasses, fatty acids, and circulating metabolites remain incompletely characterized.
Ala-Korpela, M +25 more
core +2 more sources
Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]
, 2020 Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core +1 more source
Hepatitis C virus molecular evolution: Transmission, disease progression and antiviral therapy [PDF]
, 2014 Hepatitis C virus (HCV) infection represents an important public health problem worldwide. Reduction of HCV morbidity and mortality is a current challenge owned to several viral and host factors.
Carpio Pedroza, Juan Carlos +10 more
core +1 more source
Extracellular vesicles: the next generation in gene therapy delivery.
Molecular Therapy, 2023 Extracellular vesicles (EVs) are esteemed as a promising delivery vehicle for various genetic therapeutics. They are relatively inert, non-immunogenic, biodegradable, and biocompatible.
Riccardo Cecchin +3 more
semanticscholar +1 more source
Targeting Oncogenic BRAF: Past, Present, and Future. [PDF]
, 2019 Identifying recurrent somatic genetic alterations of, and dependency on, the kinase BRAF has enabled a "precision medicine" paradigm to diagnose and treat BRAF-driven tumors.
Bivona, Trever G +2 more
core +1 more source
Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders
Frontiers in Genetics, 2019 The field of gene therapy is striving more than ever to define a path to the clinic and the market. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide.
Alireza Shahryari +8 more
semanticscholar +1 more source
Patient and Disease-Specific Induced Pluripotent Stem Cells for Discovery of Personalized Cardiovascular Drugs and Therapeutics. [PDF]
, 2020 Human induced pluripotent stem cells (iPSCs) have emerged as an effective platform for regenerative therapy, disease modeling, and drug discovery.
Chandy, Mark +2 more
core
The molecular basis for ethnic variation and histological subtype differences in prostate cancer. [PDF]
, 2013 Prostate cancer is a common malignancy among men in Western countries. Recently the morbidity and mortality of prostate cancer increase dramatically in several oriental countries including China.
Goldstein, Andrew S +2 more
core +1 more source
Genetic Variants Associated With Cancer Therapy–Induced Cardiomyopathy
Circulation, 2019 Supplemental Digital Content is available in the text.
P. García-Pavía +51 more
semanticscholar +1 more source
Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
Nature Communications, 2019 Fifty percent of inner ear disorders are caused by genetic mutations. To develop treatments for genetic inner ear disorders, we designed gene replacement therapies using synthetic adeno-associated viral vectors to deliver the coding sequence for ...
Carl Nist-Lund +11 more
semanticscholar +1 more source