Results 21 to 30 of about 1,025,168 (353)
Molecular Therapy: Methods & Clinical Development, 2019 rAAVrh74.MCK.GALGT2 is a surrogate gene therapy that inhibits muscular dystrophy in multiple animal models. Here, we report on a dose-response study of functional muscle GALGT2 expression as well as toxicity and biodistribution studies after systemic ...
Deborah A. Zygmunt +10 more
doaj +1 more source
Analysis of a conserved cellulase transcriptional regulator reveals inducer-independent production of cellulolytic enzymes in Neurospora crassa. [PDF]
, 2013 Cellulose is recalcitrant to deconstruction to glucose for use in fermentation strategies for biofuels and chemicals derived from lignocellulose. In Neurospora crassa, the transcriptional regulator, CLR-2, is required for cellulolytic gene expression and
Coradetti, Samuel +2 more
core +2 more sources
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
Efficient allelic-drive in Drosophila. [PDF]
, 2019 Gene-drive systems developed in several organisms result in super-Mendelian inheritance of transgenic insertions. Here, we generalize this "active genetic" approach to preferentially transmit allelic variants (allelic-drive) resulting from only a single ...
Berni, Mateus +9 more
core +7 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
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
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
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
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
Circumventing antivector immunity: potential use of nonhuman adenoviral vectors [PDF]
, 2014 Adenoviruses are efficient gene delivery vectors based on their ability to transduce a wide variety of cell types and drive high-level transient transgene expression. While there have been advances in modifying human adenoviral (HAdV) vectors to increase
Beard C.W. +30 more
core +3 more sources