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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. These methyl groups must be refreshed so if PRC2 is blocked the genes it has silenced. Cas9 binds and uses RNA as an address-tag.

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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells. Source link: [link]

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World AIDS Day 2023: New and Promising Treatments for HIV/AIDS

XTalks

Its mechanism of action involves blocking the protein shell (capsid) of the HIV-1 virus, disrupting multiple crucial stages of the viral lifecycle. Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR. Nearly one third of human genes lack CGIs, which would limit the use of the tool.

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2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Most antibody drugs and vaccines have been developed to target parts of the spike protein. Innovations.

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Why Women with Alzheimer’s Live Longer and Do Better Than Men with the Disease

XTalks

It helps explain why women with the disease survive longer with less severe symptoms than men during the early stages of the Alzheimer’s, despite having the same levels of toxic amyloid beta and tau proteins in their brains. The gene, called KDM6A , is a histone demethylase that is believed to function as a tumor suppressor.

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