scholarly journals Cystic fibrosis and the role of gastrointestinal outcome measures in the new era of therapeutic CFTR modulation

2015 ◽  
Vol 14 (2) ◽  
pp. 169-177 ◽  
Author(s):  
Frank A.J.A. Bodewes ◽  
Henkjan J. Verkade ◽  
Jan A.J.M. Taminiau ◽  
Drucy Borowitz ◽  
Michael Wilschanski
Genes ◽  
2021 ◽  
Vol 12 (4) ◽  
pp. 562
Author(s):  
Afsoon Sepahzad ◽  
Deborah J. Morris-Rosendahl ◽  
Jane C. Davies

Our understanding of cystic fibrosis (CF) has grown exponentially since the discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989. With evolving genetic and genomic tools, we have come to better understand the role of CFTR genotypes in the pathophysiology of the disease. This, in turn, has paved the way for the development of modulator therapies targeted at mutations in the CFTR, which are arguably one of the greatest advances in the treatment of CF. These modulator therapies, however, do not target all the mutations in CFTR that are seen in patients with CF and, furthermore, a variation in response is seen in patients with the same genotype who are taking modulator therapies. There is growing evidence to support the role of non-CFTR modifiers, both genetic and environmental, in determining the variation seen in CF morbidity and mortality and also in the response to existing therapies. This review focusses on key findings from studies using candidate gene and genome-wide approaches to identify CF modifier genes of lung disease in cystic fibrosis and considers the interaction between modifiers and the response to modulator therapies. As the use of modulator therapies expands and we gain data around outcomes, it will be of great interest to investigate this interaction further. Going forward, it will also be crucial to better understand the relative influence of genomic versus environmental factors. With this understanding, we can truly begin to deliver personalised care by better profiling the likely disease phenotype for each patient and their response to treatment.


2007 ◽  
Vol 4 (4) ◽  
pp. 370-377 ◽  
Author(s):  
N. Mayer-Hamblett ◽  
B. W. Ramsey ◽  
R. A. Kronmal

2020 ◽  
Vol 04 (03) ◽  
pp. 291-302
Author(s):  
Mariam F. Eskander ◽  
Christopher T. Aquina ◽  
Aslam Ejaz ◽  
Timothy M. Pawlik

AbstractAdvances in the field of surgical oncology have turned metastatic colorectal cancer of the liver from a lethal disease to a chronic disease and have ushered in a new era of multimodal therapy for this challenging illness. A better understanding of tumor behavior and more effective systemic therapy have led to the increased use of neoadjuvant therapy. Surgical resection remains the gold standard for treatment but without the size, distribution, and margin restrictions of the past. Lesions are considered resectable if they can safely be removed with tumor-free margins and a sufficient liver remnant. Minimally invasive liver resections are a safe alternative to open surgery and may offer some advantages. Techniques such as portal vein embolization, association of liver partition with portal vein ligation for staged hepatectomy, and radioembolization can be used to grow the liver remnant and allow for resection. If resection is not possible, nonresectional ablation therapy, including radiofrequency and microwave ablation, can be performed alone or in conjunction with resection. This article presents the most up-to-date literature on resection and ablation, with a discussion of current controversies and future directions.


2020 ◽  
Vol 5 ◽  
pp. 7-11
Author(s):  
Malek Abdel-Shehid

Calypso is a popular Caribbean musical genre that originated in the island nation of Trinidad and Tobago. The genre was developed primarily by enslaved West Africans brought to the region via the transatlantic slave trade during the seventeenth and eighteenth centuries. Although West-African Kaiso music was a major influence, the genre has also been shaped by other African genres, and by Indian, British, French, and Spanish musical cultures. Emerging in the early twentieth century, Calypso became a tool of resistance by Afro-Caribbean working-class Trinbagonians. Calypso flourished in Trinidad due to a combination of factors—namely, the migration of Afro-Caribbean people from across the region in search of upward social mobility. These people sought to expose the injustices perpetrated by a foreign European and a domestic elite against labourers in industries such as petroleum extraction. The genre is heavily anti-colonial, anti-imperial, and anti-elitist, and it advocated for regional integration. Although this did not occur immediately, Calypsonians sought to establish unity across the region regardless of race, nationality, and class through their songwriting and performing. Today, Calypso remains a unifying force and an important part of Caribbean culture. Considering Calypso's history and purpose, as well as its ever-changing creators and audiences, this essay will demonstrate that the goal of regional integration is not possible without cultural sovereignty.


2020 ◽  
Vol 10 (3) ◽  
pp. 186-193
Author(s):  
REN YANYAN ◽  

The friendship between nations lies in the mutual affinity of the people, and the people’s affinity lies in the communion of hearts. The cultural and humanities cooperation between China and Russia has a long history. In recent years, under the role of the“Belt and Road” initiative, the SCO, and the Sino-Russian Humanities Cooperation Committee, Sino-Russian culture and humanities cooperation has continued to deepen. Entering a new era, taking the opportunity to promote Sino-Russian relations into a “new era China-Russia comprehensive strategic cooperative partnership”, the development of human relations between the two countries has entered a new historical starting point, while also facing a series of problems and challenges. This article is based on the current status of Sino-Russian human relations in the new era, interprets the characteristics of Sino-Russian human relations in the new era, analyzes the problems and challenges of Sino-Russian human relations in the new era, and tries to propose solutions and solutions with a view to further developing Sino-Russian cultural and humanities relations in the new era. It is a useful reference, and provides a reference for future related research, and ultimately helps the Sino-Russian cultural and humanities relations in the new era to be stable and far-reaching.


2020 ◽  
Vol 19 ◽  
pp. S49
Author(s):  
L. Backström Eriksson ◽  
C. Laine ◽  
I. de Monestrol ◽  
A. Lindblad ◽  
E. Bergenmar-Ivarsson ◽  
...  

Antibiotics ◽  
2021 ◽  
Vol 10 (7) ◽  
pp. 766
Author(s):  
David F. Woods ◽  
Stephanie Flynn ◽  
Jose A. Caparrós-Martín ◽  
Stephen M. Stick ◽  
F. Jerry Reen ◽  
...  

The study of the respiratory microbiota has revealed that the lungs of healthy and diseased individuals harbour distinct microbial communities. Imbalances in these communities can contribute to the pathogenesis of lung disease. How these imbalances occur and establish is largely unknown. This review is focused on the genetically inherited condition of Cystic Fibrosis (CF). Understanding the microbial and host-related factors that govern the establishment of chronic CF lung inflammation and pathogen colonisation is essential. Specifically, dissecting the interplay in the inflammation–pathogen–host axis. Bile acids are important host derived and microbially modified signal molecules that have been detected in CF lungs. These bile acids are associated with inflammation and restructuring of the lung microbiota linked to chronicity. This community remodelling involves a switch in the lung microbiota from a high biodiversity/low pathogen state to a low biodiversity/pathogen-dominated state. Bile acids are particularly associated with the dominance of Proteobacterial pathogens. The ability of bile acids to impact directly on both the lung microbiota and the host response offers a unifying principle underpinning the pathogenesis of CF. The modulating role of bile acids in lung microbiota dysbiosis and inflammation could offer new potential targets for designing innovative therapeutic approaches for respiratory disease.


2021 ◽  
Author(s):  
Jeffery T. Zobell ◽  
Justin Moss ◽  
Stephanie Heuser ◽  
Lynda Roe ◽  
David C. Young

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