Off-target effect– tag –
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Drug Discovery & DDS
MDPI Accelerates Non-Coding RNA Cancer Therapy with Organ-Selective LNPs: Achieving Precise Delivery to Liver, Spleen, and Lung
MDPI Switzerland Overview Non-coding RNA (ncRNA) therapeutics in cancer are advancing, with their efficacy significantly influenced by lipid nanoparticle (LNP) characteristics like lipid composition, particle size, and surface charge. Re... -
iPS Cells & Regenerative Medicine
ARPA-H Launches AEGIS Project: Affordable Gene-Editing Therapies for Pediatric Immune Diseases via CRISPR and LNP Delivery
Princeton University USA Overview The ARPA-H THRIVE-funded AEGIS project is pioneering the development of affordable gene-editing therapies for children suffering from rare inborn errors of immunity (IEIs). By leveraging advanced CRISPR ... -
New Technology
FDA Approval a TPD Turning Point? Maria Soloveychik Cautions Single Molecule Success Doesn’t Guarantee All Targets
Drug Discovery Online USA Overview Maria Soloveychik acknowledges the FDA approval of a bivalent degrader as a crucial 'proof point' for the Targeted Protein Degradation (TPD) field but warns that this single molecular success does not n... -
New Technology
Alloy Therapeutics Unveils AntiClastic™ Cyclic RNA Platform for Enhanced Potency and Reduced Off-Target Effects in Next-Gen RNA Medicines
Alloy Therapeutics USA Overview Alloy Therapeutics introduced its AntiClastic™ Cyclic Oligonucleotide platform, which reconfigures ASOs, siRNAs, and sgRNAs into proprietary cyclic architectures. This technology aims to enhance potency an... -
New Technology
Beyond RNA: DNA-Guided CRISPR Heralds a New Era of Precision Gene Editing
Liv Hospital Turkey Overview Liv Hospital reports a significant shift in CRISPR gene editing, moving from conventional RNA-guided to potentially more stable and precise DNA-guided systems. This evolution, coupled with advancements in del... -
New Technology
First Report of CRISPR-Derived Base Editing in Human Embryos Achieves Precise DNA Changes While Avoiding Large Chromosomal Aberrations
CMN Weekly (CRISPR Medicine News) Unknown Overview CRISPR-derived base editing has been successfully applied to human embryos for the first time, achieving precise DNA changes while circumventing the large chromosomal aberrations typical... -
New Technology
Efficient Prime Editing In Vivo and In Vitro Demonstrated with Lipid Nanoparticles: High Efficacy, Low Off-Target Effects
PubMed International Overview Recent research reports the development of an optimized platform for prime editing using lipid nanoparticles (PE-LNP), demonstrating high gene editing efficiency in vivo. This LNP-based system offers a compe... -
New Technology
CRISPR Gene Editing Evolves with Cas12a2 to Shred Sick Cell DNA, Expanding Therapeutic Horizons for Cancer and Viral Infections
Top Doctor Magazine USA Overview The CRISPR gene editing landscape is rapidly evolving in 2026, driven by the FDA approval of Cas9-based CASGEVY and the discovery of the novel Cas12a2 protein's ability to 'shred' diseased cell DNA. Unlik...
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