Results 41 to 50 of about 1,410,069 (283)

Picoliter-volume inkjet printing into planar microdevice reservoirs for low-waste, high-capacity drug loading. [PDF]

open access: yes, 2017
Oral delivery of therapeutics is the preferred route for systemic drug administration due to ease of access and improved patient compliance. However, many therapeutics suffer from low oral bioavailability due to low pH and enzymatic conditions, poor ...
Bogdanoff, Derek B   +6 more
core   +1 more source

Personalized Selumetinib Dosing in Pediatric Neurofibromatosis Type 1: Insights From a Pilot Therapeutic Drug Monitoring Study

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Objective To evaluate selumetinib exposure using therapeutic drug monitoring (TDM) in pediatric patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN), assess interpatient pharmacokinetic variability, and explore the relationship between drug exposure, clinical response, and adverse effects.
Janka Kovács   +8 more
wiley   +1 more source

Targeting HDAC6 to treat heart failure with preserved ejection fraction in mice

open access: yesNature Communications
Heart failure with preserved ejection fraction (HFpEF) poses therapeutic challenges due to the limited treatment options. Building upon our previous research that demonstrates the efficacy of histone deacetylase 6 (HDAC6) inhibition in a genetic ...
Sara Ranjbarvaziri   +18 more
doaj   +1 more source

Posttranslational and Therapeutic Control of Gasdermin-Mediated Pyroptosis and Inflammation

open access: yesFrontiers in Immunology, 2021
Pyroptosis is a proinflammatory form of cell death, mediated by membrane pore-forming proteins called gasdermins. Gasdermin pores allow the release of the pro-inflammatory cytokines IL-1β and IL-18 and cause cell swelling and cell lysis leading to ...
Fabian A. Fischer   +2 more
doaj   +1 more source

Current applications and future potential for bioinorganic chemistry in the development of anticancer drugs [PDF]

open access: yes, 2009
This review illustrates notable recent progress in the field of medicinal bioinorganic chemistry as many new approaches to the design of innovative metal-based anticancer drugs are emerging.
Aird   +75 more
core   +1 more source

AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models

open access: yesNature Communications
Hypertrophic cardiomyopathy (HCM) affects approximately 600,000 people in the United States. Loss-of-function mutations in Myosin Binding Protein C3, MYBPC3, are the most common genetic cause of HCM, with the majority of mutations resulting in ...
Amara Greer-Short   +29 more
doaj   +1 more source

A Comprehensive Overview of circRNAs: Emerging Biomarkers and Potential Therapeutics in Gynecological Cancers

open access: yesFrontiers in Cell and Developmental Biology, 2021
Circular RNA (circRNA) is a highly conserved, stable and abundant non-coding RNA (ncRNA). Also, some circRNAs play an essential part in the progression of human cancers. CircRNA is different from traditional linear RNA.
Yalan Ma   +5 more
doaj   +1 more source

Fluorescence-based quantification of messenger RNA and plasmid DNA decay kinetics in extracellular biological fluids and cell extracts [PDF]

open access: yes, 2020
Extracellular and intracellular degradation of nucleic acids remains an issue in non-viral gene therapy. Understanding biodegradation is critical for the rational design of gene therapeutics in order to maintain stability and functionality at the target ...
Braeckmans, Kevin   +6 more
core   +1 more source

Sirolimus for Extracranial Arteriovenous Malformations: A Scoping Review of the Evidence in Syndromic and Non‐Syndromic Cases

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Arteriovenous malformations (AVMs) are rare, high‐flow, vascular anomalies that can occur either sporadically or as part of a genetic syndrome. AVMs can progress with serious morbidity and even mortality if left unchecked. Sirolimus is an mTOR inhibitor that is effective in low‐flow vascular malformations; however, its role in AVMs is unclear.
Will Swansson   +3 more
wiley   +1 more source

WRN inhibition leads to its chromatin-associated degradation via the PIAS4-RNF4-p97/VCP axis

open access: yesNature Communications
Synthetic lethality provides an attractive strategy for developing targeted cancer therapies. For example, cancer cells with high levels of microsatellite instability (MSI-H) are dependent on the Werner (WRN) helicase for survival.
Fernando Rodríguez Pérez   +25 more
doaj   +1 more source

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