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New whitepaper on trends in precision medicine: Cancer

Drug Discovery World

Precision medicine uses information about an individual’s genes, proteins, and environment to prevent, diagnose, and treat diseases, including cancer. Differences in DNA, RNA, and protein can be identified in individual tissue samples, and even in individual cells.

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Evolution in Hepatitis B and C Virus: Can we expect success similar to Hepatitis C in Hepatitis B as well?

Delveinsight

The first approved treatment for hepatitis C was a series of protein-based injections called recombinant interferon-alfa (IFNa). Interferons are naturally occurring proteins in the body; recombinant IFNa is the protein-based drug that works to mobilize the body’s natural immune system to fight disease. in January 2019.

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RNA editing oligonucleotide selected for AATD treatment 

Drug Discovery World

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.

RNA 52
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The doors CRISPR libraries have and will open in phenotypic drug screening 

Drug Discovery World

Bacterial RNA is replaced by a synthetic guide RNA (gRNA) that can recognise a specific target DNA sequence. These modifications lead to alterations in the function of the proteins expressed by these genes, manifesting in cellular processes dependent on those proteins. CRISPR libraries: Why do we need them?

Genome 52
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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

Oxlumo works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.

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Image-based screening of pooled genetic libraries

Drug Discovery World

A high-content imaging assay can be tailored to the biological questions under investigation, enabling in-depth characterisation of complex cellular phenotypes such as protein/RNA localisation, metabolic state, cell morphology, cell-cell interactions, and live-cell dynamics.