Remove DNA Remove Protein Expression Remove Small Molecule
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The Future of Drug Discovery: Tackling the Undruggable with New Biotechnologies

DrugBank

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

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CRISPR and single-cell sequencing highlight genetic variants for traits and diseases

Drug Target Review

The researchers turned to CRISPR —a gene editing tool that uses “molecular scissors to cut DNA ,” according to Sanjana—to edit the regions identified by GWAS. The researchers then use CRISPR to target each of the regions of the genomes implicated by GWAS and conduct single-cell sequencing to evaluate gene and protein expression.

Disease 52
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Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

Several viral vectors are being used currently, in addition to non-viral vectors, such as oligonucleotides, naked DNA, and lipoplexes and polyplexes. They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify protein expression.