Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo ...
Incyte will share updated clinical single-cell data characterizing its calreticulin-targeted therapy for myeloproliferative ...
Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in ...
Sofi-cel is an allogeneic, off-the-shelf, CD7-targeted CAR-T cell therapy being developed for T-cell cancers. Allotera uses ...
A mutation-agnostic CRISPR-Cas strategy that inserts a functional rhodopsin coding sequence into the human RHO locus has preserved retinal structure, function and visual acuity for a full year in a ...
Base editing offers far more versatility than its name implies. The technique was initially developed to correct single-nucleotide variants by replacing one DNA base with another. However, researchers ...
CRISPR Activation Reduces Arterial Lipids in Mice A nuclease-inactive CRISPR-Cas9 system increased endogenous expression of two protective HDL components without cutting genomic DNA. In a small mouse ...
Ole Bock moderated two sessions at the Chinese Society of Cell & Gene Therapy Annual Meeting in Beijing, and CMN received the ...
YOLT-204 is an investigational off-the-shelf, in vivo gene-editing therapy for transfusion-dependent β-thalassemia. It uses YolTech’s proprietary lipid nanoparticle delivery system to deliver a gene ...
AX-0811 is an investigational RNA-editing oligonucleotide therapy being developed for cholestatic liver diseases. The therapy uses ProQR’s Axiomer RNA-editing platform to edit RNA encoding NTCP ...
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