CSL3 is a novel cationic switchable lipid, pH-triggered molecular
switch for siRNA delivery.In vivo factor VII silencing efficiency of the CSL3-based formulations
was compared to that of the CSL4-based formulation
(Fig. 5E). CSL3-based LNP were found to demonstrate a
dose–response knockdown, resulting in significant silencing
efficacy (∼50% silencing) at 1 mg kg−1.These results confirm the crucial role of the
pH-sensitive conformational change of CSL3 in the in vivo
transfection efficacy, in agreement with the in vitro results. The
in vivo silencing efficacy of the CSL3 LNP may be further
improved by lowering the molar percentage of DMG-PEG2000 in
the formulation11,33 or by using combinatorial approaches to
enhance the potency of the CSL3 lipid structure as well as the
blood stability of the LNP (currently under investigation).
Cat.No
DC81075
Name
CSL3
Chemical Properties
CAS
2607971-24-2
Formula
C48H77N3O2
MW
728.15
Storage
References
switch for siRNA delivery-Nanoscale, 2017, 9, 31
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