Abstract：
:Since the discovery of the CF gene over a decade ago, several groups worldwide have explored the potential of gene therapy, the insertion of a normal copy of the gene into the respiratory epithelium. Both viral and synthetic gene transfer agents have been designed to this end, although problems with repeat application have occurred with the former have occurred. Here, we will briefly review success in clinical trials to date. Cell therapy, replacement of the CF cell with a healthy, normal cell, is at a much earlier stage of development, studies mainly being preclinical or observational. Some evidence of plasticity of, for example, bone marrow cells, has been demonstrated, although for the airway, levels of correction appear to be extremely low and a degree of damage seems to be a prerequisite. Whether this approach will be able to achieve clinical success remains to be seen.
journal_name
Paediatr Respir Revjournal_title
Paediatric respiratory reviewsauthors
Davies JCdoi
10.1016/j.prrv.2006.04.214subject
Has Abstractpub_date
2006-01-01 00:00:00pages
S163-5eissn
1526-0542issn
1526-0550pii
S1526-0542(06)00228-4journal_volume
7 Suppl 1pub_type
杂志文章,评审abstract：:The aim of this study was to review and synthesize the existing knowledge of the effects of ultrafine particles [UFPs] with a specific focus on children's health. An extensive literature search identified 16 studies fulfilling the criteria set for the review. One of the most important findings of the review was that, ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2019.06.003
更新日期：2019-11-01 00:00:00
abstract：:Sudden unexpected death is one of the most frequent ways of dying in the first year of life after the neonatal period. It is however, much less frequent after the first birthday. Investigations into the cause of death are very important, for a significant proportion of these sudden deaths can be explained only after a...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2009.12.002
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journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1053/prrv.2000.0009
更新日期：2000-03-01 00:00:00
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journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2016.03.002
更新日期：2016-09-01 00:00:00
abstract：:Since preschool wheezing is the common expression of several heterogeneous disorders, identification of children at risk for persistent asthma is particularly challenging. To date, efforts to predict the outcome of preschool wheeze have mainly relied on predictive rules consisting of simple clinical and laboratory par...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2012.08.004
更新日期：2013-03-01 00:00:00
abstract：:Bronchopulmonary dysplasia (BPD) is a common complication of preterm birth. Chest imaging is important in making the diagnosis of BPD, and in assessing for complications. More recently computerised tomography (CT) scanning has provided insights in to the pathophysiology of BPD. Studies in infants, young and school age...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2010.05.005
更新日期：2010-09-01 00:00:00
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journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1053/prrv.2002.0180
更新日期：2002-03-01 00:00:00
abstract：:The dynamic interaction between the heart and lungs leads to a degree of respiratory co-morbidity including both restrictive and obstructive airway abnormalities, which may be overlooked in children with congenital and acquired heart disease. The improving imaging techniques of the heart, both foetal and post-natal co...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2007.08.006
更新日期：2007-12-01 00:00:00
abstract：:Severe acute respiratory syndrome (SARS) is a newly described respiratory infection with pandemic potential. The causative agent is a new strain of coronavirus most likely originating from wild animals. This disease first emerged in November 2002 in Guangdong Province, China. Early in the outbreak the infection had be...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2004.07.011
更新日期：2004-12-01 00:00:00
abstract：:Asthma is most frequent chronic disease in childhood. Childhood asthma frequently persists to adulthood and the available evidence suggests that the severity of childhood asthma predicts the severity of asthma in the adulthood. To prevent long term remodeling of the airways and improve prognosis, early diagnosis and t...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2006.04.171
更新日期：2006-01-01 00:00:00
abstract：:The anatomical and functional development of the lung appears especially vulnerable to a whole range of insults during gestation and the first few years of life. A significant proportion of adult lung disease originates in utero or early infancy. Most publications on this topic are descriptive retrospective studies. A...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/s1526-0542(02)00311-1
更新日期：2003-03-01 00:00:00
abstract：:The Clinical and Functional Translation of CFTR (CFTR2) project presents a novel approach to clinical and functional annotation of mutations identified in disease-causing genes. Phenotype and genotype information on approximately 40,000 cystic fibrosis (CF) patients were collected from registries and large clinics. Th...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章
doi：10.1016/j.prrv.2013.01.006
更新日期：2013-05-01 00:00:00
abstract：:Renal disease is relatively rare in cystic fibrosis even though the cystic fibrosis transmembrane regulator (CFTR) protein is expressed in abundance in the kidney. Aberrant CFTR expression probably explains the subtle abnormalities in renal concentrating and diluting ability described in cystic fibrosis and possibly t...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章
doi：
更新日期：2002-06-01 00:00:00
abstract：:The major morbidity and mortality associated with cerebral palsy (CP) relates to respiratory compromise. This manifests through repeated pulmonary aspiration, airway colonization with pathogenic bacteria, the evolution of bronchiectasis and sleep disordered breathing. An accurate assessment involving a multidisciplina...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2008.10.003
更新日期：2009-03-01 00:00:00
abstract：:Air pollution exposure has increased extensively in recent years and there is considerable evidence that exposure to particulate matter can lead to adverse respiratory outcomes. The health impacts of exposure to air pollution during the prenatal period is especially concerning as it can impair organogenesis and organ ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2016.08.008
更新日期：2017-01-01 00:00:00
abstract：:Respiratory viruses cause a number of clinical 'syndromes' in the intensive care unit with different viruses being able to produce similar clinical pictures. Our main presenting problems are upper airway (e.g. croup and tracheitis), lower airway with intrapulmonary shunt (e.g. bronchitis and pneumonia), lower airway w...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：
更新日期：2003-09-01 00:00:00
abstract：:Pneumonia is highly prevalent in both developed and developing countries. In this review we list the main organisms affecting children with pneumonia and we propose a summary of the best possible diagnostic and therapeutic measures. ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2006.04.221
更新日期：2006-01-01 00:00:00
abstract：:Primary ciliary dyskinesia (PCD), also known as immotile-cilia syndrome, is a rare genetic disease that is inherited in an autosomal recessive manner. Several studies have explored certain aspects of PCD in the Arab world, yet much is still lacking in terms of identifying the different characteristics of this disease....
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2018.09.002
更新日期：2019-02-01 00:00:00
abstract：:Cystic fibrosis related diabetes (CFRD) is a common complication of cystic fibrosis, caused by a fall in insulin secretion with age in individuals with pancreatic insufficiency. CFRD is associated with worse clinical status and increased mortality. Treatment of CFRD with insulin results in sustained improvements in lu...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章
doi：10.1016/j.prrv.2013.02.002
更新日期：2013-05-01 00:00:00
abstract：:Polysomnography is an elaborate diagnostic test composed of numerous data-collecting sensors working concomitantly to aid in the evaluation of varied sleep disorders in all age groups. Polysomnography is the study of choice for the assessment of pediatric sleep-disordered breathing, including obstructive sleep apnea s...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2019.09.009
更新日期：2020-04-01 00:00:00
abstract：:Allergic bronchopulmonary aspergillosis (ABPA) is a severe complication in children, adolescents and adults with cystic fibrosis (CF), the prevalence of which ranges from 6-25%. The disease is the result of the colonisation of the respiratory tract by fungi of the genus Aspergillus, commonly Aspergillus fumigatus, and...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2005.09.003
更新日期：2006-03-01 00:00:00
abstract：:During the first year of life and particularly the first 6 months autonomic control of the cardio-respiratory system is still undergoing maturation and infants are at risk of cardio-respiratory instability. These instabilities are most marked during sleep, which is important as infants spend the majority of each 24 ho...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2013.02.012
更新日期：2014-06-01 00:00:00
abstract：:The range of techniques available to assess respiratory muscles weakness has greatly expanded over recent years. Respiratory muscle tests can be divided into non-invasive tests, such as lung function parameters, maximal static pressures and sniff nasal pressure and invasive tests, such as transdiaphragmatic pressures ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/s1526-0542(03)00051-4
更新日期：2003-09-01 00:00:00
abstract：:Accumulating data suggest that prenatal compromises leading to intrauterine growth restriction (IUGR) increase the risk for respiratory deficiencies after birth. In this respect, a growing body of epidemiological evidence in infants, children and adults indicates that small for gestational (SGA) birth weight can adver...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2012.10.001
更新日期：2013-12-01 00:00:00
abstract：:Vocal cord dysfunction is characterised by paradoxical vocal cord adduction that occurs during inspiration, resulting in symptoms of dyspnoea, wheeze, chest or throat tightness and cough. Although the condition is well described in children and adults, confusion with asthma often triggers the use of an aggressive trea...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2007.05.004
更新日期：2007-06-01 00:00:00
abstract：:Control of the cardiovascular and respiratory systems undergoes rapid maturation during infancy. Sleep is at a lifetime maximum during this period and has a marked influence on cardiorespiratory function. The mechanisms leading to sudden infant death syndrome (SIDS) may include a failure in the neural integration of t...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2004.04.011
更新日期：2004-09-01 00:00:00
abstract：:There are multiple aetiologies for childhood bronchiectasis unrelated to cystic fibrosis. Some of these aetiologies, such as those predisposing to recurrent lung infections, e.g. immunodeficiencies, require treatment of the underlying condition and disease-specific pulmonary pathogens within the airway. Regardless of ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章
doi：10.1016/j.prrv.2010.10.012
更新日期：2011-06-01 00:00:00
abstract：:Asthma is a heterogeneous disease and it is therefore unrealistic to expect that inhaled corticosteroids (ICS) would be appropriate first line preventer therapy for all children with asthma. There is good theoretical and clinical trial evidence demonstrating that leukotriene receptor antagonists (LTRAs) are more effec...
journal_title：Paediatric respiratory reviews
pub_type： 评论,杂志文章
doi：10.1016/j.prrv.2011.05.008
更新日期：2011-12-01 00:00:00
abstract：:Chylothorax is the accumulation of chyle in the pleural space, as a result of damage to the thoracic duct. Chyle is milky fluid enriched with fat secreted from the intestinal cells and lymphatic fluid. Chylothorax in children, is most commonly seen as a complication of cardiothoracic surgery but may occur in newborns ...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2009.06.008
更新日期：2009-12-01 00:00:00
abstract：:Whilst substantial progress has been made in the treatment of cystic fibrosis, the disease still carries a significant burden in terms of symptoms, requirement for treatment and early mortality. The last decade has witnessed a new era in the development of small molecule drugs targeting the CFTR protein, which for the...
journal_title：Paediatric respiratory reviews
pub_type： 杂志文章,评审
doi：10.1016/j.prrv.2017.03.008
更新日期：2018-03-01 00:00:00
