Publications by authors named "D K Sahel"
Article Synopsis
- CRISPR/Cas acts like molecular scissors for precise gene editing using mechanisms like non-homologous end joining (NHEJ) or homology-directed repair (HDR), with various Cas effectors available for different applications.
- SpCas9 is the most studied version, recognized as a breakthrough for correcting genetic mutations, while newer variants like Cas12 and Cas13 are noted for their specific abilities to recognize and cleave target nucleic acids.
- The combination of CRISPR technology, diagnostics, and nanotechnology has led to the development of advanced biosensing systems capable of providing quantitative fluorescent signals for diagnostic purposes.
Article Synopsis
- Delivering macromolecules through the skin is challenging due to the skin's barrier, but various methods have been explored to overcome this, including the use of nanocarriers.
- A cationic lipo-polymeric nanocarrier was developed to successfully deliver a model protein, showing optimal sizes and zeta potentials that indicate suitability for skin application.
- After testing, the nanocarrier demonstrated a promising skin permeation rate, low toxicity, and effective delivery of protein therapeutics, suggesting a viable approach for transdermal delivery.
We present an efficient approach for synthesizing cationic poly(ethylene imine) derivatives using the multicomponent split-Ugi reaction to rapidly create a library of complex functional ionizable lipopolymers. We synthesized a diverse library of 155 polymers, formulated them into polyplexes to establish structure-activity relationships crucial for endosomal escape and efficient transfection. After discovering a lead structure, lipopolymer-lipid hybrid nanoparticles are introduced to preferentially deliver to and elicit effective mRNA transfection in lung endothelium and immune cells, including T cells with low toxicity.
View Article and Find Full Text PDFEye-related diseases, specifically retinal dystrophy (RD) conditions, are the leading cause of blindness worldwide. Gene addition, regulation, or editing could potentially treat such diseases through gene expression regulation. CRISPR/Cas9 gene editing is one of the most prominent and precise gene editing tools which could be employed to edit genes related to the dystrophic condition.
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