Annals of the Child Neurology SocietyBackground Pathogenic CYFIP2 variants cause developmental and epileptic encephalopathy (DEE), characterized by early‐onset intractable epilepsy and developmental delay. The disease course has not been delineated.
Michaela Squire +3 more
doaj +1 more source
Correction: Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models. [PDF]
Gene Ther, 2023 Chilcott EM +3 more
europepmc +1 more source
Genetic basis of phage-host interaction: Towards effective phage therapy of non-typhoidal Salmonella [PDF]
, 2022 Non-typhoidal Salmonella (NTS) have adapted to cause invasive illness in humans. Bacteria have developed MDR against current antibiotics. Bacteriophage therapy is the hope for bacterial treatment however one of the key limitations is the limited host ...
Mohammed, M.
core
Genetic Therapy for Intervertebral Disc Degeneration. [PDF]
Int J Mol Sci, 2021 Roh EJ +7 more
europepmc +1 more source
GENETIC SPECTRUM OF PRIMARY DYSLIPIDEMIAS IN CHILDREN - SINGLE CENTER EXPERIENCE [PDF]
Primary dyslipidemias are heterogenous metabolic disorders caused by pathogenic genetic variants. Over 100 genes have been identified that impact lipid metabolism, with familial hypercholesterolemia being the most common form, occurring in the general ...
Sarajlija, Adrijan +8 more
core
A 10-Year Review on Advancements in Identifying and Treating Intellectual Disability Caused by Genetic Variations [PDF]
Intellectual disability (ID) is a prevalent neurodevelopmental disorder characterized by neurodevelopmental defects such as the congenital impairment of intellectual function and restricted adaptive behavior.
Kexin Hou, Xinyan Zheng
core +1 more source
Genetic Mechanisms of Asthma and the Implications for Drug Repositioning [PDF]
, 2018 Asthma is a chronic disease that is caused by airway inflammation. The main features of asthma are airway hyperresponsiveness (AHR) and reversible airway obstruction. The disease is mainly managed using drug therapy.
Yue Huo, Hong-Yu Zhang
core +1 more source
Gene therapy by hepatocyte growth factor results in regression of experimental liver fibrosis
Российский журнал гастроэнтерологии, гепатологии, колопроктологии, 2010 Aim of investigation. Studying of efficacy of genetic therapy of liver fibrosis at mice by human hepatocyte growth factor (HGF) and evaluation of potentials of hydroporation method for delivery of genetic complexes to the liver.Material and methods ...
N. A. Dzhoyashvili +7 more
doaj
Molecular Therapy: Nucleic Acids, 2016 Usher syndrome (USH) is the most common cause of combined deaf-blindness in man. The hearing loss can be partly compensated by providing patients with hearing aids or cochlear implants, but the loss of vision is currently untreatable.
Radulfus WN Slijkerman +15 more
doaj +1 more source
Genetic Therapy and Molecular Targeted Therapy in Oncology: Safety, Pharmacovigilance, and Perspectives for Research and Clinical Practice. [PDF]
Int J Mol Sci, 2022 Orzetti S +8 more
europepmc +1 more source