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Gene editing: beyond the hype

pharmaphorum

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.

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As Vertex and CRISPR build out Casgevy launch, doctor sees strong demand and warns of possible supply hitches

Fierce Pharma

As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their CRISPR-based gene-editing therapy Casgevy, one doctor who treats sickle cell disease patients is already | As Vertex Pharmaceuticals and CRISPR Therapeutics lay the groundwork for the launch of their world-first CRISPR-based gene therapy Casgevy, one doctor (..)

Doctors 103
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Lipid nanoparticles and mRNA used to treat hereditary blindness

Drug Discovery World

The study, led by OSU Associate Professor of Pharmaceutical Sciences Gaurav Sahay, Oregon State doctoral student Marco Herrera-Barrera, and Oregon Health & Science University Assistant Professor of Ophthalmology Renee Ryals, was published in Science Advances. .

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Meet the researcher: Dr Stephen Jones

Drug Discovery World

At the moment, we are focused on gene editing tools, and specifically CRISPR nucleases, trying to design strategies that allow us to use them more safely and effectively. Our goal is to develop strategies that allow us to understand how they work and how they find their targets, so we can hopefully improve them.

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Stem cell therapy ‘cures’ first woman with HIV, say researchers

pharmaphorum

Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32 Sangamo was working on a treatment based on autologous (patient-derived) stem cells and T cells that were gene-edited to carry the CCR5?32

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IWD 2024: Eight women taking the lead in drug discovery

Drug Discovery World

This inspired her to change direction, first by investigating the role of Epstein-Barr Virus in epithelial cancers during her PhD, and subsequently TGFβ-signalling in breast cancer during her post-doctoral career. Read the interview.

Drugs 69
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Advancements in upstream CLD for enhanced biotherapeutics production

Drug Discovery World

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.