CFTR (CF Transmembrane Conductance Regulator)

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.

Finally, we review the changes due to treatment with CFTR modulators and their possible implications for future screening and monitoring strategies. As people with CF enjoy better quality of life and improved survival, screening for CF-related morbidities will be evolving constantly.

We identified seven potential pain modifiers in CF, including chymotrypsin C (CTRC), serine protease inhibitor Kazal-Type 1 (SPINK1), tumour necrosis factor (TNF), ATP-binding cassette subfamily B Member 1 (ABCB1), protease serine 1 (PRSS1) and transforming growth factor beta 1 (TGFB1) interacting with the CFTR gene...This in silico analysis highlights potential genes and biochemical pathways implicated in pain pathways that could significantly impact pain perception in people living with CF and their response to prescribed therapies. Further functional analyses are needed to include CF participants and provide a physiological relevance on how genetic polymorphisms of identified GMs may impact their pain phenotype or profile.

The overall prevalence of CFTR PVs in people with solid tumors was higher than expected. These results suggest that carriers of CFTR PV may benefit from enhanced cancer screening.

SSLs showed a mixed acidic mucin phenotype and downregulated epithelial bicarbonate transporters. This may impair mucin expansion and hydration, leading to the formation of adhesive mucous caps, showing the potential link between defective bicarbonate transport and mucin physiology in SSLs.

The role of concomitant sodium-potassium ATPase inhibition is explained. Therapeutic interventions including already known CFTR inhibitors for excessive fluid secretion in the upper respiratory tract and remedies for inflammation-induced fluid accumulation in the lower respiratory tract due to CFTR dysfunction using anti-inflammatory and CFTR activating agents are introduced.