Results 301 to 310 of about 1,593,615 (383)

Mitochondria‐Targeted Delivery of siRNA by Amphiphilic Nanovectors Self‐Assembled from a Cationic [4]Helicene‐Squalene Ester

open access: yesChemBioChem, EarlyView.
A small amphiphilic helicene effectively complexes siRNA, leading to nanoassemblies which transport siRNA to the mitochondria where it silences the protein expression of the mitochondria‐encoded cytochrome c oxidase subunit 1 gene. The active delivery of therapeutic nucleic acids with subcellular precision is an open challenge.
Lamiaa M. A. Ali   +7 more
wiley   +1 more source

Messenger RNA-encoded antibody approach for targeting extracellular and intracellular tau. [PDF]

open access: yesBrain Commun
Wongsodirdjo P   +6 more
europepmc   +1 more source

Citrus Flavonoids for Cystic Fibrosis Treatment

open access: yesChemBioChem, EarlyView.
Studies conducted since the late 1990s show substantial activity of selected citrus flavonoids as cystic fibrosis transmembrane conductance regulator (CFTR) membrane protein activators. The thesis of this work is that aptly formulated citrus flavonoids hold sigificant potential for developing a multitarget treatment of cystic fibrosis combining in a ...
Mario Pagliaro   +5 more
wiley   +1 more source

Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates. [PDF]

open access: yesGut
Córdoba KM   +29 more
europepmc   +1 more source

A Phase 1, Randomized, Open‐Label, Parallel Group Study to Evaluate the Relative Bioavailability and Safety of Subcutaneous Bepirovirsen when Delivered from a Vial or Prefilled Syringe Fitted with a Safety Syringe Device in Healthy Adult Participants

open access: yesClinical Pharmacology in Drug Development, EarlyView.
Abstract Bepirovirsen, an antisense oligonucleotide in development for the treatment of chronic hepatitis B virus (HBV) infection, is administered from glass vials as a subcutaneous (SC) injection by healthcare professionals (HCPs). A ready‐to‐use prefilled syringe (PFS) assembled with a safety syringe device (SSD) has been developed to make ...
Amir S. Youssef   +15 more
wiley   +1 more source

Advancing Predictions of Oral Drug Absorption, CYP3A4 Induction, and Transporter‐Mediated Interactions Using a Human Primary Intestinal 3D Model (EpiIntestinal™)

open access: yesClinical Pharmacology &Therapeutics, EarlyView.
Accurate prediction of oral drug absorption in humans is essential for early drug development; however, physiologically relevant human models are lacking. This study aims to comprehensively assess the EpiIntestinal™, a human primary intestinal 3D model, for its ability to predict oral absorption (Fa), intestinal availability (Fg), CYP3A4 induction, and
Paresh P. Chothe   +4 more
wiley   +1 more source

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