CFTR (CF Transmembrane Conductance Regulator)

Methylation of adenosine at the N6 position (m6A modification) is the most prevalent epitranscriptomic modification in mammals, regulating RNA stability, splicing, translation, and decay. Moreover, knockdown of the erasers fat mass and obesity-associated protein (FTO) or AlkB homolog 5 as well as treatment with entacapone, an FTO inhibitor, increased PTX sensitivity in PTX-resistant A549 cells. These results suggest that the m6A-dependent regulation of MRP7 expression contributes to PTX resistance, highlighting a potential therapeutic avenue in lung cancer.

Moreover, these variants could act as preliminary biomarker candidates for clinical validation. The obtained knowledge gained from the current research could eventually assist in disease diagnosis and therapy design.

The role of CFTR modulator therapy perinatally continues to be of great interest, related to both the mother with CF as well as the infant in utero and after birth. As the median life expectancy for PwCF continues to increase, consideration must also be given for treatment of cardiovascular disease and other comorbidities as well as cancer screening and other preventative care measures.

Difluprednate showed the highest pharmacovigilance signal (ROR: 963.67; 95% CI: 316.27-2936.31; n = 4). Notably, CFTR modulators exhibited striking signals: ivacaftor (ROR: 30.75; 95% CI: 18.06-52.37; n = 14), elexacaftor-ivacaftor-tezacaftor (ROR: 15.58; 95% CI: 9.86-24.63; n = 19), and ivacaftor-lumacaftor (ROR: 13.2; 95% CI: 7.9-22.07; n = 15). This study provides a comprehensive large-scale pharmacovigilance profile of drug-induced pediatric cataracts, identifying agents with high-risk pharmacovigilance signals and underscoring the need for proactive ocular monitoring. These findings can inform clinical decision making and prevention strategies and guide future mechanistic research.

Treatment with octreotide, omeprazole, and pancreatic enzyme replacement therapy led to symptom resolution...The clinical manifestations of CF are diverse, and digestive tract symptoms are common; therefore, early identification and diagnosis are required. As chr7: 117250723 G > T may be a pathogenic gene, long-term follow-up is needed.

There is a gap in healthcare for pwCF, especially for those who do not routinely interact with a PCP. CF clinicians may not be up to date on primary care management and PCPs may not be comfortable with treating patients with a high acuity chronic condition. PCPs should be better integrated into the CF care team to ensure pwCF are receiving comprehensive care.

P4, N=33, Completed, University of Alabama at Birmingham | Recruiting --> Completed | N=25 --> 33

While cisplatin-based chemoradiotherapy regimens (gemcitabine-cisplatin [GP] and docetaxel-cisplatin-5-fluorouracil [TPF]) remain standard treatments for advanced nasopharyngeal carcinoma (NPC), 30-40% of patients exhibit intrinsic chemoresistance, resulting in therapeutic failure. Notably, Spautin-1, a potent USP10 inhibitor, demonstrates synergistic therapeutic activity with cisplatin in diminished tumor growth and metastasis in NPC mice. This research established the USP10-MRPS7/MRPS23-β-catenin axis as a promising precision medicine strategy to combat metastatic dissemination and reverse cisplatin chemoresistance in advanced NPC, which offers a promising opportunity to develop cisplatin sensitizers for the clinical translation of NPC therapies.

BSHX may improve assisted reproductive technology pregnancy rates by regulating sex hormones and uterine fluid balance to prevent COH-induced endometrial damage.

SBP ameliorates pyrotinib-induced diarrhea by modulating the EGF-cAMP-CFTR signaling axis, thereby inhibiting intestinal chloride secretion and restoring electrolyte balance. In addition to confirming its therapeutic effect, this study reveals a previously unrecognized mechanism whereby SBP downregulates EGFR-dependent CFTR activation, offering a mechanistic explanation for its antidiarrheal properties. These findings not only highlight the mechanistic novelty of SBP in the context of targeted-therapy-associated diarrhea but also provide a scientific foundation supporting its clinical application and future translational development.

This review synthesizes current understanding of SPEM's pathogenesis, molecular mechanisms, and clinical implications. Furthermore, it proposes integration of SPEM assessment into gastric cancer risk stratification protocols to enable earlier intervention strategies that interrupt carcinogenesis at its most vulnerable and potentially reversible stage.

In this Review, we summarize current knowledge on the epidemiology, pathophysiology, diagnosis and treatment of CFRD, CFBD, growth and puberty, hypogonadism and infertility, iatrogenic adrenal insufficiency and perimenopause in patients with cystic fibrosis. We also consider future research priorities in the field.