Results 21 to 30 of about 1,021,897 (307)
Molecular Therapy: Methods & Clinical Development, 2018 Recombinant adeno-associated virus (rAAV)rh74.MCK.GALGT2 is a muscle-specific gene therapy that is being developed to treat forms of muscular dystrophy.
Rui Xu +17 more
doaj +1 more source
Gene Therapy for Cardiovascular Disease [PDF]
, 2003 The last decade has seen substantial advances in the development of gene therapy strategies and vector technology for the treatment of a diverse number of diseases, with a view to translating the successes observed in animal models into the clinic ...
Baker, Andrew H. +3 more
core +4 more sources
Viral gene drive in herpesviruses [PDF]
Nature Communications, 2019 AbstractHerpesviruses are ubiquitous pathogens in need of novel therapeutic solutions. Current engineered gene drive strategies rely on sexual reproduction, and are thought to be restricted to sexual organisms. Here, we report on the design of a novel gene drive system that allows the spread of an engineered trait in populations of DNA viruses and, in ...
Walter, Marius, Verdin, Eric
openaire +3 more sources
The T-cell oncogene Tal2 is a Target of PU.1 and upregulated during osteoclastogenesis [PDF]
, 2013 Transcription factors play a crucial role in regulating differentiation processes during human life and are important in disease. The basic helix-loop-helix transcription factors Tal1 and Lyl1 play a major role in the regulation of gene expression in the
Courtial, Nadine +5 more
core +6 more sources
A multiple-instance scoring method to predict tissue-specific cis-regulatory motifs and regions [PDF]
, 2009 Transcription is the central process of gene regulation. In higher eukaryotes, the transcription of a gene is usually regulated by multiple cis-regulatory regions (CRRs).
Jin Gu
core +2 more sources
Neurobiology of Disease, 2010 Vaccination has become an important therapeutic approach to the treatment of Alzheimer's disease (AD), however, immunization with Aβ amyloid can have unwanted, potentially lethal, side effects.
Chiou-Miin Wang +4 more
doaj +1 more source
Local adaptation with high gene flow: temperature parameters drive adaptation to altitude in the common frog (Rana temporaria) [PDF]
, 2014 Both environmental and genetic influences can result in phenotypic variation. Quantifying the relative contributions of local adaptation and phenotypic plasticity to phenotypes is key to understanding the effect of environmental variation on populations.
Biek, R. +3 more
core +2 more sources
Principles for gene drive research [PDF]
Science, 2017 Sponsors and supporters of gene drive research respond to a National Academies ...
Claudia, Emerson +3 more
openaire +2 more sources
Kinase activity is required for the toxic effects of mutant LRRK2/dardarin
Neurobiology of Disease, 2006 Mutations in the LRRK2 gene, coding for dardarin, cause dominantly inherited Parkinson's disease (PD). Dardarin is a large protein, and mutations are found throughout the gene including the kinase domain.
Elisa Greggio +17 more
doaj +1 more source
A confinable home-and-rescue gene drive for population modification
eLife, 2021 Homing-based gene drives, engineered using CRISPR/Cas9, have been proposed to spread desirable genes throughout populations. However, invasion of such drives can be hindered by the accumulation of resistant alleles.
Nikolay P Kandul +4 more
doaj +1 more source