Niosome-based approach for in situ gene delivery to retina and brain cortex as immune-privileged tissues

N Al Qtaish, I Gallego, I Villate-Beitia, M Sainz-Ramos… - Pharmaceutics, 2020 - mdpi.com
Non-viral vectors have emerged as a promising alternative to viral gene delivery systems
due to their safer profile. Among non-viral vectors, recently, niosomes have shown favorable …

The protective A673T mutation of amyloid precursor protein (APP) in Alzheimer's disease

Q Xia, XY Yang, JB Shi, ZJ Liu, YH Peng… - Molecular …, 2021 - Springer
Alzheimer's disease is a progressive neurodegenerative disorder characterized by
extracellular amyloid beta peptides and neurofibrillary tangles consisted of intracellular …

Patisiran in hATTR amyloidosis: six-month latency period before efficacy

L Gentile, M Russo, M Luigetti, G Bisogni… - Brain Sciences, 2021 - mdpi.com
Hereditary amyloidosis associated with mutations in the transthyretin gene (hATTR) is a
progressive devastating disease, with a fatal outcome occurring within 10years after onset …

Small Interfering RNAs Containing Dioxane- and Morpholino-Derived Nucleotide Analogues Show Improved Off-Target Profiles and Chirality-Dependent In Vivo …

A Hofmeister, K Jahn-Hofmann, B Brunner… - Journal of Medicinal …, 2022 - ACS Publications
To expand the applicability of recently developed dioxane-and morpholino-based
nucleotide analogues, their seed region destabilizing properties in small interfering RNAs …

Molecular elements: novel approaches for molecular building

R Wang, X Wang, S Xie, Y Zhang… - … of the Royal …, 2023 - royalsocietypublishing.org
Classically, a molecular element (ME) is a pure substance composed of two or more atoms
of the same element. However, MEs, in the context of this review, can be any molecules as …

Sixteen different types of lipid-conjugated siRNAs containing saturated and unsaturated fatty acids and exhibiting enhanced RNAi potency

T Kubo, Y Nishimura, Y Sato, K Yanagihara… - ACS chemical …, 2020 - ACS Publications
SiRNAs are strong gene-silencing agents that function in a target sequence-specific
manner. Although siRNAs might one day be used in therapy for intractable diseases such as …

Survivin silencing improved the cytotoxicity of carboplatin and melphalan in Y79 and primary retinoblastoma cells

VP Gibson, RM Derbali, HT Phan, H Tahiri… - International Journal of …, 2020 - Elsevier
Survivin stands out as one of the most specific cancer targets discovered to date. Although
single inhibition, eg through small interfering RNA (siRNA), has shown modest results in …

Gene Therapy for Inherited Metabolic Diseases

N Longo, K Lai - Nutrition Management of Inherited Metabolic Diseases …, 2022 - Springer
Gene-based therapies, defined as the transfer of genetic materials to treat a disease, can
potentially restore the function of the missing enzyme in individuals with inborn errors of …

Syntheses of morpholine-based nucleotide analogs for hepatic siRNA targeting and stabilization

A Hofmeister, K Jahn-Hofmann, B Brunner… - Journal of Medicinal …, 2021 - ACS Publications
A morpholine-based nucleotide analog was developed as a building block for hepatic
siRNA targeting and stabilization. Attachment of an asialoglycoprotein-binding GalNAc …

Investigation of pH-sensitive mechanism and anticancer application of switchable lipid nanoparticles

V Passos Gibson - 2020 - papyrus.bib.umontreal.ca
Cationic switchable lipids belong to the class of stimuli-responsive materials. When
incorporated in lipid nanoparticles (LNP), switchable LNP promote pH-triggered delivery of …