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What single cells are revealing about brain disorders

Drug Target Review

From maths and physics to molecular biology Schnall-Levin did not begin in the life sciences. “I I started looking around and getting interested in biology. I got my eyes opened to molecular biology, bioinformatics, computational biology and these emerging fields at the time.”

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Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig

Alta Sciences

Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig aasimakopoulos Thu, 07/10/2025 - 08:00 I recently returned from attending the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. WHO WAS AT ASGCT 2025?

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A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays. Visit our website to get a copy.

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Levers for Biological Progress

Codon

I’ve chosen these two because I think they are the linchpin by which we’ll be able to build broadly useful AI models for cell and molecular biology. Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1.

DNA 132
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Scientists discover network of cells and genes involved in Crohn’s disease complication

Broad Institute

The scientists suggest that new therapies that target these genes could directly address fibrosis and potentially be more effective for this complication than existing drugs, which are primarily focused on reducing inflammation. But now we can potentially think about additive therapies that more directly target the fibrotic process.”

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Machine learning guides researchers to new synthetic genetic switches

Broad Institute

In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. This limits our ability to design gene therapies that only effect certain cell types in the human body." Chan School of Public Health. "By

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Unlocking Undruggable Targets: Shifting Paradigms in Modern Drug Discovery

DrugBank

But as molecular biology has advanced, so too has our approach to finding new drugs. Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. This method was more about serendipity than science.