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Gene Targeting in Disease Networks [PDF]

open access: yesFrontiers in Genetics, 2021
Profiling of whole transcriptomes has become a cornerstone of molecular biology and an invaluable tool for the characterization of clinical phenotypes and the identification of disease subtypes. Analyses of these data are becoming ever more sophisticated
Deborah Weighill   +9 more
doaj   +2 more sources

Measles virus glycoprotein-based lentiviral targeting vectors that avoid neutralizing antibodies [PDF]

open access: yes, 2012
Lentiviral vectors (LVs) are potent gene transfer vehicles frequently applied in research and recently also in clinical trials. Retargeting LV entry to cell types of interest is a key issue to improve gene transfer safety and efficacy.
Abel, Tobias   +5 more
core   +11 more sources

CRISPR/Cas9-Mediated Multi-Allelic Gene Targeting in Sugarcane Confers Herbicide Tolerance

open access: yesFrontiers in Genome Editing, 2021
Sugarcane is the source of 80% of the sugar and 26% of the bioethanol produced globally. However, its complex, highly polyploid genome (2n = 100 – 120) impedes crop improvement. Here, we report efficient and reproducible gene targeting (GT) in sugarcane,
Mehmet Tufan Oz   +6 more
doaj   +1 more source

Constitutive and conditional gene knockout mice for the study of intervertebral disc degeneration: Current status, decision considerations, and future possibilities

open access: yesJOR Spine, 2023
There have been an increasing number of patients with degenerative disc diseases due to the aging population. In light of this, studies on the pathogenesis of intervertebral disc degeneration have become a hot topic, and gene knockout mice have become a ...
Ze‐Yu Lu   +6 more
doaj   +1 more source

Leucine-Rich Repeats and Transmembrane Domain 2 Controls Protein Sorting in the Striatal Projection System and Its Deficiency Causes Disturbances in Motor Responses and Monoamine Dynamics

open access: yesFrontiers in Molecular Neuroscience, 2022
The striatum is involved in action selection, and its disturbance can cause movement disorders. Here, we show that leucine-rich repeats and transmembrane domain 2 (Lrtm2) controls protein sorting in striatal projection systems, and its deficiency causes ...
Misato Ichise   +11 more
doaj   +1 more source

Therapeutic gene targeting [PDF]

open access: yesGene Therapy, 1998
Gene targeting is the use of homologous recombination to make defined alterations to the genome. One of the possible outcomes of gene targeting is the accurate correction of genetic defects, and this would make it the ideal method of gene therapy for single gene disorder.
R J, Yáñez, A C, Porter
openaire   +2 more sources

Gene targeting in plants [PDF]

open access: yesThe EMBO Journal, 1987
Although the generation of transgenic plants is now routine, the integration of foreign genetic information has so far been at random sites in the genome. We now present evidence for directed integration into a predicted location in the host plant genome.
J, Paszkowski   +3 more
openaire   +2 more sources

T-Gene: improved target gene prediction [PDF]

open access: yesBioinformatics, 2019
Abstract Motivation Identifying the genes regulated by a given transcription factor (TF) (its ‘target genes’) is a key step in developing a comprehensive understanding of gene regulation. Previously, we developed a method (CisMapper) for predicting the target genes of a TF based solely on the ...
Timothy O’Connor   +3 more
openaire   +4 more sources

Rhes depletion promotes striatal accumulation and aggregation of mutant huntingtin in a presymptomatic HD mouse model

open access: yesFrontiers in Aging Neuroscience, 2023
IntroductionHuntington’s disease (HD) is caused by CAG trinucleotide repeats in the HTT gene. Selective neurodegeneration in the striatum is prominent in HD, despite widespread expression of mutant HTT (mHTT).
Yongcheng Pan   +9 more
doaj   +1 more source

Targeting Epilepsy Genes [PDF]

open access: yesNeuron, 1996
In medieval art it was traditional to show a demon emanating from a suffering epileptic at the conclusion of a seizure, with concern clearly evident regarding its inevitable reappearance. In this century, anticonvulsant drugs have been developed for their ability to control, rather than prevent or reverse, seizure disorders.
openaire   +2 more sources

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