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Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
Li, Bowen ; Manan, Rajith Singh ; Liang, Shun-Qing , et al. Nat. Biotechnol.,2023,41(10):1410-1415. DOI: 10.1038/s41587-023-01679-x PubMed ID: 36997680
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Abstract: The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen a combinatorial library of biodegradable ionizable lipids to build inhalable delivery vehicles for mRNA and CRISPR-Cas9 gene editors. Lead lipid nanoparticles are amenable for repeated intratracheal dosing and could achieve efficient gene editing in lung epithelium, providing avenues for gene therapy of congenital lung diseases.
Purchased from AmBeed: 14916-80-4 ; 294-90-6 ; 13093-04-4 ; 65604-89-9 ; 22366-98-9 ; 143-28-2 ; 1484-84-0 ; 112-92-5 ; 3433-37-2 ; 34803-66-2 ; 622-26-4 ; 934-98-5 ; 3529-08-6 ; 123-70-6 ; 23356-96-9 ; 534-26-9 ; 4730-54-5 ; 108-00-9 ; 51388-00-2 ; 6711-48-4 ; 506-43-4 ; 2038-03-1 ; 142-25-6 ; 27578-60-5 ; 67980-77-2 ; 4572-03-6 ; 14156-95-7 ; 10563-26-5 ; 4097-88-5 ; 111-33-1 ; 123-12-6 ; 6261-22-9 ; 496808-04-9 ; 3644-18-6 ; 764-60-3 ; 1002-36-4 ; 51-45-6 ; 112086-54-1 ; 22104-79-6 ; 67529-83-3 ; 10563-29-8 ; 294-90-6 ; 506-43-4 ; 20739-58-6 ; 13901-38-7 ; 938459-02-0 ; 7209-38-3 ; 51721-39-2 ; 18128-28-4 ; 105-83-9 ; 877-96-3 ; 14712-23-3 ; 915922-79-1 ; 205059-32-1 ; 5298-72-6 ; 22763-69-5 ...More
CAS No. : | 7209-38-3 | MDL No. : | MFCD00006161 |
Formula : | C10H24N4 | Boiling Point : | - |
Linear Structure Formula : | (CH2CH2N(CH2CH2CH2NH2))2 | InChI Key : | XUSNPFGLKGCWGN-UHFFFAOYSA-N |
M.W : | 200.32 | Pubchem ID : | 81629 |
Synonyms : |
|
Chemical Name : | 3,3'-(Piperazine-1,4-diyl)bis(propan-1-amine) |
Signal Word: | Danger | Class: | 8 |
Precautionary Statements: | P280-P305+P351+P338-P310 | UN#: | 2735 |
Hazard Statements: | H314 | Packing Group: | Ⅱ |
GHS Pictogram: |
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