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Scientists engineer CRISPR enzymes that evade the immune system

Broad Institute

Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies. Lisa Yang Center for Autism Research at MIT, and the K.

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Researchers use machine learning to engineer 'bespoke enzymes' for gene editing

Science Daily: Pharmacology News

A new paper showcases the power of scalable protein engineering combined with machine learning to boost progress in the field of gene and cell therapy. Genome editing has advanced at a rapid pace with promising results for treating genetic conditions -- but there is always room for improvement.

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Chimeric deubiquitinase engineering reveals structural basis for specific inhibition of the mitophagy regulator USP30

Covalent Modifiers

Here we report the crystal structure of human USP30 in complex with a specific inhibitor, enabled by chimeric protein engineering. Collectively, our work establishes a generalizable chimeric protein-engineering strategy to aid deubiquitinase crystallization and enables structure-based drug design with relevance to neurodegeneration.

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Ultrafast 12-minute MRI maps brain chemistry to spot disease before symptoms

Science Daily: Pharmacology News

Illinois engineers fused ultrafast imaging with smart algorithms to peek at living brain chemistry, turning routine MRIs into metabolic microscopes. The system distinguishes healthy regions, grades tumors, and forecasts MS flare-ups long before structural MRI can. Precision-medicine neurology just moved closer to reality.

Disease 227
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Site-Specific Molecular Glues for the 14-3-3/Tau pS214 ProteinProtein Interaction via Reversible Covalent Imine Tethering

Covalent Modifiers

Exploiting the unique topologies and functionalities of the different binding sites enabled the engineering of selectivity for this initial molecular glue matter for the pS214 binding site, over a second 14-3-3 binding site in Tau (pS324).

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Actithera draws new investors to radiopharma drug pitch

BioPharma Drive: Drug Pricing

The technology works by attaching a radioisotope to a targeting compound via a specially engineered “linker” molecule. Dive Insight: Radiopharmaceuticals offer a better way to target tumor-killing radiation more precisely to cancerous cells and, accordingly, they’ve drawn rising interest from drugmakers.

Drugs 155
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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases. we are first focused on engineering tRNAs to restore protein production in diseases caused by premature termination codons.

Disease 80