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What single cells are revealing about brain disorders

Drug Target Review

Neurological diseases are among the most complex and least understood conditions in medicine. Despite the knowledge that certain genes increase the risk of contracting diseases like Alzheimer’s and Parkinson’s, it is still not fully understood how those diseases develop in the brain. But that is beginning to change.

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Why Mallory Freeberg is creating a perturbation catalogue

The Open Targets Blog

You can also link the consequences of the perturbation to disease symptoms, suggesting novel targets for drug discovery. Connecting targets to disease, and predicting the impact of perturbing genes therapeutically will contribute to the development of new therapies. Perturbation Catalogue home page How will researchers use your data?

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Genomic score predicts patients' progression to multiple myeloma

Broad Institute

The score, called an MM-like score, assesses the severity of disease and risk of progression to active cancer, with higher scores indicating faster progression. In the future, the MM-like score could be used in clinical practice to inform decisions about early intervention. The study was published in Nature Genetics.

DNA
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Scientific workflow for hypothesis testing in drug discovery: Part 1

Drug Target Review

Without this information, the interpretation of the dataset may be speculative or lead to inaccurate conclusions. These linked materials may describe the cell lines used, the specific conditions under which experiments were performed, and any potential limitations of the data.

RNA
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Key projects

Broad Institute

From foundational multimodal models to targeted disease prediction, phenotyping, and drug repurposing, ML4H combines large-scale data, deep clinical insight, and cutting-edge AI methods to drive innovation across cardiology, neurology, and beyond.

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From siloed data to breakthroughs: multimodal AI in drug discovery

Drug Target Review

NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. This approach overcomes the limitations of traditional methods that analyse only single modalities of information. This shift is driven by open access policies to biomedical data and the advent of next-generation sequencing (NGS).

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Therapeutic Oligos 2025 Keynote Speakers Announced

Elrig

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

RNA