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Gene Targeting

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Transcriptomes of lineage-specific Drosophila neuroblasts profiled by genetic targeting and robotic sorting.

Development (Cambridge, England)
A brain consists of numerous distinct neurons arising from a limited number of progenitors, called neuroblasts in Drosophila. Each neuroblast produces a specific neuronal lineage. To unravel the transcriptional networks that underlie the development ...

miRAW: A deep learning-based approach to predict microRNA targets by analyzing whole microRNA transcripts.

PLoS computational biology
MicroRNAs (miRNAs) are small non-coding RNAs that regulate gene expression by binding to partially complementary regions within the 3'UTR of their target genes. Computational methods play an important role in target prediction and assume that the miR...

SiRNA silencing efficacy prediction based on a deep architecture.

BMC genomics
BACKGROUND: Small interfering RNA (siRNA) can be used to post-transcriptional gene regulation by knocking down targeted genes. In functional genomics, biomedical research and cancer therapeutics, siRNA design is a critical research topic. Various com...

An overview and metanalysis of machine and deep learning-based CRISPR gRNA design tools.

RNA biology
The CRISPR-Cas9 system has become the most promising and versatile tool for genetic manipulation applications. Albeit the technology has been broadly adopted by both academic and pharmaceutic societies, the activity (on-target) and specificity (off-t...

Machine learning discriminates a movement disorder in a zebrafish model of Parkinson's disease.

Disease models & mechanisms
Animal models of human disease provide an system that can reveal molecular mechanisms by which mutations cause pathology, and, moreover, have the potential to provide a valuable tool for drug development. Here, we have developed a zebrafish model of...

A deep learning approach to identify gene targets of a therapeutic for human splicing disorders.

Nature communications
Pre-mRNA splicing is a key controller of human gene expression. Disturbances in splicing due to mutation lead to dysregulated protein expression and contribute to a substantial fraction of human disease. Several classes of splicing modulator compound...

Uncertainty-aware and interpretable evaluation of Cas9-gRNA and Cas12a-gRNA specificity for fully matched and partially mismatched targets with Deep Kernel Learning.

Nucleic acids research
The choice of guide RNA (gRNA) for CRISPR-based gene targeting is an essential step in gene editing applications, but the prediction of gRNA specificity remains challenging. Lack of transparency and focus on point estimates of efficiency disregarding...