Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases

Camacho, Manuel V.; Kong, Qingzhong

doi:10.1007/978-3-031-20565-1_36

Cited by 1 publication

(1 citation statement)

References 68 publications

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“…Increasing evidence suggests that gene therapy has the potential to treat a wide range of ailments as a therapeutic agent [ 28 , 29 , 30 , 31 ]. The major challenge in gene therapy is the need for a vector to deliver the gene at the target site due to its vulnerability to enzymatic degradation.…”

Section: Discussionmentioning

confidence: 99%

Non-Invasive Intranasal Delivery of pApoE2: Effect of Multiple Dosing on the ApoE2 Expression in Mice Brain

Gothwal,

Lamptey,

Trivedi

et al. 2023

IJMS

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Chitosan-based polymeric micelles are promising non-viral nanocarriers for safe and targeted gene delivery. Multi-functionalized chitosan polymeric micelles were prepared by grafting fatty acid, cell-penetrating peptide, and mannose on the chitosan backbone. The polymeric micelles were subjected to surface morphology and surface topography using scanning electron microscopy and atomic force microscopy, respectively. The hemotoxic profile of the prepared polymeric micelles was established against erythrocytes and was found to be <5% hemotoxic up to the concentration of 600 µg/mL. In vitro ApoE2 expression in primary astrocytes and neurons was analyzed. Multi-functionalized polymeric micelles produced greater (p < 0.05) transfection in astrocytes and neurons in comparison to mono-functionalized micelles. Intranasal administration of polymeric micelles/pApoE2 polyplex led to significantly higher (p < 0.05) in vivo pApoE2 expression than chitosan and unfunctionalized polymeric micelles-treated mice groups. The outcomes of this study predict that the developed multi-functionalized polymeric micelles could be an effective and safe gene delivery platform to the brain through the intranasal route.

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