2014 Conference Access Set (Plenaries, Symposia & Workshops)


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S08: CFTR: Gene Editing Strategies for Therapy & Research

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S08: CFTR: Gene Editing Strategies for Therapy & Research

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will describe two technologies to manipulate genes such as CFTR for potential therapeutic purposes as well as for more fundamental mechanistic studies about cell biology. One of these technologies is genome editing by the CRISPR/Cas9 system, in which targeted alterations are made in the genome of particular cells. Various applications of the technology, from mutation correction in primary cells to creation of mutations to study a mutation's effects on cellular processes or as a therapeutic tool will be discussed. RNA editing, by which one can correct mutations in messenger RNA without modifying the genomic DNA from which it was transcribed will also be discussed.

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S09: GMS: The Complex Genetics of CF & Their Functional Effects

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S09: GMS: The Complex Genetics of CF & Their Functional Effects

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Recent research has identified modifier genes of CF disease severity through genome-wide association studies, linkage analysis studies and candidate gene approaches. This session will describe several studies focusing on how to understand the biological and functional significance of several of these modifier genes.

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S10: NT: Fruit of the Pipeline: The Latest Clinical Trial Data

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S10: NT: Fruit of the Pipeline: The Latest Clinical Trial Data

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will address recent clinical trials that test novel approaches to CF therapies directed at overcoming the basic defect of missing or dysfunctional CFTR.

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S11: INF/MIC: Multiple Inhaled Antibiotics: Multiple Therapeutic Considerations

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S11: INF/MIC: Multiple Inhaled Antibiotics: Multiple Therapeutic Considerations

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Suppressive antibiotic treatment of CF patients with chronic P. aeruginosa airway infection is the standard of care in Europe and North America. With the recent inhaled aztreonam approval, there are now three antipseudomonal antibiotic classes that are readily available and commonly utilized to manage chronic P. aeruginosa airway infections: polymyxins (colistimethate), aminoglycosides (gentamicin, tobramycin) and beta lactams (aztreonam). These agents have all been studied and developed as monotherapies, but there is growing interest in the use of these agents in various combinations in individual patients to improve outcomes and extend the period of efficacy of suppressive treatment.

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S12: CLIN: Caroline McPherson Symposium: Care of Infants & Pre-school-aged Children With CF

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S12: CLIN: Caroline McPherson Symposium: Care of Infants & Pre-school-aged Children With CF

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

Early aggressive treatment of pulmonary disease and optimization of nutritional status in cystic fibrosis during early childhood is essential to achieving improved outcomes in adulthood. Unfortunately, there are several challenges within this age group ranging from the extensive need for parental education, rapid movement through developmental stages and lack of concrete evidence from randomized controlled trials to guide care. This session will address the infant and preschool guidelines in the context of this challenging developmental period of life.

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S13: SW/PSYCH: CFF/ECFS Recommendations of the International Guidelines Committee on Mental Health in CF

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S13: SW/PSYCH: CFF/ECFS Recommendations of the International Guidelines Committee on Mental Health in CF

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

CFF & ECFS have sponsored an International Guidelines Committee on mental health in CF. This session will present the proposed guidelines on screening patients and caregivers for depression and anxiety. Our proposed recommendations include screening individuals with CF ages 12 through adulthood and caregivers of children with CF birth to 17 years. We have recommended specific screening tools, as well as evidence-based psychological and pharmacological interventions. We will present the results of an international survey of health care providers which asked about current screening practices, availability of mental health professionals, and infrastructure needs for implementation.

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S14: NTR: The Relationship of Nutrition to Quality of Life

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S14: NTR: The Relationship of Nutrition to Quality of Life

Oct 10, 2014 10:30am ‐ Oct 10, 2014 12:20pm

This session will review current knowledge of the relationship of nutrition to Quality of Life; specifically weight, height, body image, supplemental feeding, newborn screening and the feeding relationship, and nutrient intake and depression

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S15: APP&D: CF Airway Inflammation & Therapeutic Targets

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S15: APP&D: CF Airway Inflammation & Therapeutic Targets

Oct 11, 2014 10:30am ‐ Oct 11, 2014 12:20pm

This symposium begins with an update on the inflammatory response in the CF lung. Airway epithelial and immune cells contribute to inflammation in CF. There is a large influx of neutrophils that release damaging mediators including proteases and oxygen radicals. The second talk focuses on the importance of cellular immunity in CF respiratory pathophysiology, which has only recently begun to be understood. It is well known that nitric oxide (NO) is decreased in CF exhaled gas. The significance of this and the use of NO as a therapy is discussed in the third talk. The final talk reviews the role of anti-inflammatory therapy in CF and provides an update on clinical trials in this area.

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S16: NT-CFTR: Treating Every Patient: Theratype Approaches to CF Intervention

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S16: NT-CFTR: Treating Every Patient: Theratype Approaches to CF Intervention

Oct 11, 2014 10:30am ‐ Oct 11, 2014 12:20pm

The session will focus on new therapeutic approaches for CF patients carrying non-F508 mutations. Mutations have traditionally been grouped by their according to type (nonsense, missense, etc.) or by the CFTR functional classes. However, in the era of novel therapies it may be more useful to characterize mutations in terms of their response to particular therapies. We will discuss the CFTR2 project and how it has defined the genetic epidemiology across North America and Europe. We will also introduce therapeutic approaches for patients carrying nonsense and splicing mutations as well as the option of clinical trials for patients carrying rare or private mutations.

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S17: INF/MIC: Omics of CF Airway Infection

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S17: INF/MIC: Omics of CF Airway Infection

Oct 11, 2014 10:30am ‐ Oct 11, 2014 12:20pm

‘Omics” methods extensively characterize clinical specimens. The data from these increasingly sophisticated techniques can be correlated with clinical outcomes to identify candidate disease biomarkers and mechanisms, using intensive computational analytical methods. Recent discoveries using these tools have already changed our understanding of CF airway infection and inflammation. In this session, we will review the historical origins and recent progress in the study of the CF airway using these ‘omics approaches. We will discuss how, by characterizing the CF airway microbiome, transcriptome, metabolome and inflammasome, these tools are providing fresh insights into CF lung disease.

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