Results 111 to 120 of about 47,774 (288)

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

open access: yesBiomedicines, 2017
Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with ...
Philippine Aupy   +3 more
doaj   +1 more source

Bioconjugate Strategies for Antisense Therapeutic Delivery to Glioblastoma Stem Cells

open access: yes, 2020
Antisense therapeutics, including antisense oligonucleotides (AONs) and small interfering ribonucleic acids (siRNAs), are powerful tools for regulating genes, making them a promising therapy for diseases such as cancer where oncogenic genes are over ...
Arnold, Amy Elizabeth
core   +1 more source

A comparative study of cationic formulations for the delivery of siRNA and DNA

open access: yes, 2009
RNA interference (RNAi) provides a specific and efficient way to silence gene expression; therefore, it is an attractive tool to be used in basic research on gene function as well as gene therapy.
Kwok, N.P.A.
core  

Ursolic Acid Alleviates Orchitis via Modulating Ferroptosis in Peritubular Myoid Cells of Testis

open access: yesAnimal Research and One Health, EarlyView.
Ursolic acid modulates key ferroptosis‐related pathways in the peritubular myoid cells of the testis in forest musk deer. ABSTRACT Chronic stress adversely affects reproductive health, increasing susceptibility to pathologies such as orchitis. Additionally, the maintenance of iron homeostasis is essential for preserving the function of the testicles ...
Yang Wang   +11 more
wiley   +1 more source

CNS delivery of antisense oligodeoxynucleotides using biodegradable microspheres [PDF]

open access: yes
Antisense oligodeoxynucleotides can selectively inhibit gene expression provided they are delivered to their target site successfully for a sufficient duration. Biodegradable microspheres have previously been developed for the potential systemic delivery
Smith, Dawn L.
core  

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić   +4 more
wiley   +1 more source

026 | Graphene oxide nanosystem for antisense miRNA-21 delivery in hepatocellular carcinoma

open access: yesJournal of Biological Research
Nanotechnology-based strategies for targeted drug delivery have attracted considerable interest, particularly in gene regulation therapies (Di Stefano, 2023).
Società Italiana di Biologia Sperimentale
doaj  

Therapeutic and diagnostic applications of antisense peptide nucleic acids

open access: yesMolecular Therapy: Nucleic Acids
Peptide nucleic acids (PNAs) are synthetic nucleic acid analogs with a neutral N-(2-aminoethyl) glycine backbone. PNAs possess unique physicochemical characteristics such as increased resistance to enzymatic degradation, ionic strength and stability over
Victoria MacLelland   +2 more
doaj   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić   +4 more
wiley   +1 more source

Polycationic graft copolymers as carriers for delivery of antisense oligonucleotides

open access: yes, 2002
Polycationic graft copolymers as carriers for delivery of antisense ...
Koňák, Čestmír
core  

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