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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CHARMs are an elegant solution to the problem of silencing disease genes, and they have the potential to have an important position in the future of genetic medicines.” “The

Disease 142
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SARS-CoV-2: advancing the production of the ACE2 protein

Drug Target Review

To find a DNA fragment that contained the intact ACE2 gene, complete with its embedded regulatory information, they searched libraries of cloned DNA fragments generated as part of the Human Genome Project. The team used the intact human ACE2 gene to increase the chances that mouse cells would incorporate and read the gene correctly.

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Addressing increasingly resistant drugs by infectious agents

Drug Target Review

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

Drugs 111
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Marvin Caruthers receives inaugural Merkin Prize in ceremony at the Broad Institute for DNA synthesis technology

Broad Institute

Related links Merkin Prize Inaugural Merkin Prize in Biomedical Technology awarded to Dr. Marvin Caruthers for developing technology that efficiently synthesizes DNA The inaugural Richard N. Caruthers was announced as the winner in June for his development, in 1981, of an efficient, automated technology for synthesizing DNA.

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What Biology Can Learn from Physics

Codon

Predictive Models A “ sequence-to-function ” predictive model – an algorithm that determines a protein’s likely function solely by looking at the DNA sequence encoding it – is the natural successor to AlphaFold2. A predictive model for sequence-to-expression would raise the “hit” rates.

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The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

Conversations in Drug Development Trends

Heterogeneity in Hematological Cancers Due to their disease heterogeneity, hematological cancers are well-suited for personalized approaches. Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways.

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

Drug Target Review

These studies are conducted by comparing the genomes of large populations to find variants that occur more often in those with a specific disease or trait. These studies are conducted by comparing the genomes of large populations to find variants that occur more often in those with a specific disease or trait. “A

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