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 …
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 …
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 …
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 …
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 …
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 …
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
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 …
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 …
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 …
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 …
incorporated in lipid nanoparticles (LNP), switchable LNP promote pH-triggered delivery of …