Delivery of PAMAM dendrimers and dendriplexes across natural barriers (blood-brain barrier and placental barrier) in healthy pregnant mice.

ABSTRACT: Gene therapy is an important tool for treating fetal diseases that allows for the delivery and integration of therapeutic genes into the genome of cells carrying mutations. Nanomolecules, like PAMAM dendrimers, have recently come into wider use for carrying vectors as they have several advantages over viral vectors. Namely, (1) tunable size and surface chemistry, (2) uniform size, (3) the ability to target specific tissues, and (4) the ability to carry large biomolecules and drugs. Recently, we demonstrated that 4th generation (G4) PAMAM dendrimer with a cystamine core and a non-toxic surface having 90% -OH and 10% -NH₂ groups (D-Cys) could cross the blood-brain barrier following injection into the bloodstream. In the current study, as a proof of concept, we delivered the dendri

SUBMITTER: Kuhn E 

PROVIDER: S-EPMC11393257 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature