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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. The chemical modifications that regulate gene activity are called epigenetic markers. Cas9 binds and uses RNA as an address-tag.

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New gene-editing tool is more accurate than CRISPR

Drug Discovery World

Scientists at the University of Sydney have developed a gene-editing tool with greater accuracy and flexibility than CRISPR. SeekRNA uses a programmable ribonucleic acid (RNA) strand that can directly identify sites for insertion in genetic sequences, simplifying the editing process and reducing errors.

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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. Experts speak DDW asked a range of drug discovery experts where they thought the drug discovery expertise happening in the UK.

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Casting aside CRISPR scissors and making a point with base editors

pharmaphorum

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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

Drug Discovery World

The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. Bacterial RNA is replaced by a synthetic guide RNA (gRNA) that can recognise a specific target DNA sequence. CRISPR libraries: Why do we need them?

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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Worldwide Clinical Trials

Tofersen (Intrathecal injection) This is an antisense oligonucleotide (ASO) that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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