Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
Cells, 2023 Despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on ...
S. Issa +3 more
semanticscholar +1 more source
Hepatitis C virus pharmacogenomics in Latin American populations: implications in the era of direct-acting antivirals [PDF]
, 2017 In recent years, great progress has been made in the field of new therapeutic options for hepatitis C virus (HCV) infection. The new direct-acting antiviral agents (DAAs) represent a great hope for millions of chronically infected individuals because ...
Caputo, Mariela +4 more
core +1 more source
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
New England Journal of Medicine, 2019 Genome sequencing is often pivotal in the diagnosis of rare diseases, but many of these conditions lack specific treatments. We describe how molecular diagnosis of a rare, fatal neurodegenerative condition led to the rational design, testing, and ...
Jinkuk Kim +52 more
semanticscholar +1 more source
Tumor cell plasticity in targeted therapy-induced resistance: mechanisms and new strategies
Signal Transduction and Targeted Therapy, 2023 Despite the success of targeted therapies in cancer treatment, therapy-induced resistance remains a major obstacle to a complete cure. Tumor cells evade treatments and relapse via phenotypic switching driven by intrinsic or induced cell plasticity ...
Zhenjun Shi +8 more
semanticscholar +1 more source
Genes and primary headaches: discovering new potential therapeutic targets [PDF]
, 2013 Genetic studies have clearly shown that primary headaches (migraine, tension-type headache and cluster headache) are multifactorial disorders characterized by a complex interaction between different genes and environmental factors.
De Martino, Paola +8 more
core +3 more sources
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
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
Non-immune fetal hydrops: etiology and outcome according to gestational age at diagnosis. [PDF]
, 2020 OBJECTIVE: Fetal hydrops is associated with increased perinatal morbidity and mortality. The etiology and outcome of fetal hydrops may differ according to the gestational age at diagnosis.
A. Bhide +12 more
core +2 more sources
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