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Chemical Structure| 2036272-55-4 Chemical Structure| 2036272-55-4
Chemical Structure| 2036272-55-4

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ALC-0315, a cationic lipid, is a potent siRNA delivery vehicle. It can be used to form LNPs.

4.5 *For Research Use Only !

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Product Citations

Product Citations

Onuma, Haruno ; Shimizu, Rina ; Suzuki, Yuichi ; Sato, Mina ; Harashima, Hideyoshi ; Sato, Yusuke

Abstract: The delivery of the CRISPR/Cas ribonucleoprotein (RNP) has received attention for clinical applications owing to its high efficiency with few off-target effects. Lipid nanoparticles (LNPs) are potential non-viral vectors for the delivery of RNPs. Herein, we report the engineering of a branched scaffold structure of ionizable lipids for hepatic delivery of RNPs. Both the total carbon number and branching position were critical for the functional delivery of RNPs. The optimal ionizable lipid exhibited a more than 98% reduction in transthyretin protein after a single dose with no obvious signs of toxicity. Mechanistic study has revealed that optimal LNPs have a unique “flower-like structure” that depends on both the lipid structure and the payload and that these LNPs accumulate in hepatocytes in an apolipoprotein E-independent manner. These results represent a major step toward the realization of in vivo genome editing therapy via RNP delivery using chemically synthesizable LNP formulations.

Keywords: genome editing ; ribonucleoprotein ; lipid nanoparticles ; ionizable lipid ; scaffold ; liver

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Product Details of ALC-0315

CAS No. :2036272-55-4
Formula : C48H95NO5
M.W : 766.27
SMILES Code : O=C(OCCCCCCN(CCCCO)CCCCCCOC(=O)C(CCCCCC)CCCCCCCC)C(CCCCCC)CCCCCCCC
MDL No. :MFCD33402175
InChI Key :QGWBEETXHOVFQS-UHFFFAOYSA-N
Pubchem ID :122666778

Safety of ALC-0315

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H315-H319
Precautionary Statements:P264-P280-P302+P352-P337+P313-P305+P351+P338-P362+P364-P332+P313
 

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