A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines
Adv Drug Deliv Rev. 2024 Apr 14:115305. doi: 10.1016/j.addr.2024.115305. Online ahead of print.ABSTRACTCystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative gene delivery strategies to address the mutations in the gene. This review delves into the promises and challenges of non-viral gene delivery for CF therapy and explores strategies to overcome these hurdles. Several emerging technologies and nucleic acid cargos for CF gene therapy are discussed. Novel formulation approaches including lipid and polymeric nanoparticles promise enhanced delivery through the CF mucus barrier, augmentin...
Source: Advanced Drug Delivery Reviews - April 16, 2024 Category: Drugs & Pharmacology Authors: Namratha Turuvekere Vittala Murthy Kseniia Vlasova Jonas Renner Antony Jozic Gaurav Sahay Source Type: research
A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines
Adv Drug Deliv Rev. 2024 Apr 16;209:115305. doi: 10.1016/j.addr.2024.115305. Online ahead of print.ABSTRACTCystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative gene delivery strategies to address the mutations in the gene. This review delves into the promises and challenges of non-viral gene delivery for CF therapy and explores strategies to overcome these hurdles. Several emerging technologies and nucleic acid cargos for CF gene therapy are discussed. Novel formulation approaches including lipid and polymeric nanoparticles promise enhanced delivery through the CF mucus barrier, augme...
Source: Advanced Drug Delivery Reviews - April 16, 2024 Category: Drugs & Pharmacology Authors: Namratha Turuvekere Vittala Murthy Kseniia Vlasova Jonas Renner Antony Jozic Gaurav Sahay Source Type: research
News article
Persistence and evolution of Pseudomonas aeruginosa following initiation of highly effective modulator therapy in cystic fibrosis. With the widespread availability of elexacaftor/tezacaftor/ivacaftor (ETI) and dramatic improvement in respiratory symptoms, attention is turning to the role of therapies designed to maintain lung health. Some early studies have reported a reduced bacterial burden in the lungs after ETI, though results are easily confounded by the reduction in sputum samples and the impact on chronic P. aeruginosa infection remains unknown. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 16, 2024 Category: Respiratory Medicine Source Type: research
Inquilinus limosus, an atypical Gram-negative rod from lung transplant recipients and cystic fibrosis patients
Ann Biol Clin (Paris). 2024 Apr 1;82(1):0. doi: 10.1684/abc.2024.1876. Online ahead of print.NO ABSTRACTPMID:38616576 | DOI:10.1684/abc.2024.1876 (Source: Annales de Biologie Clinique)
Source: Annales de Biologie Clinique - April 15, 2024 Category: Biochemistry Authors: Eric Farfour St éphane Corvec Thomas Guillard H élène Revillet Source Type: research
Parental perceptions of body weight and appetite in infants and toddlers with cystic fibrosis
Appetite. 2024 Apr 13:107357. doi: 10.1016/j.appet.2024.107357. Online ahead of print.ABSTRACTNutritional status has clinical relevance and is a target of guidance to parents of children with cystic fibrosis (CF). Growth is routinely monitored in CF clinics but there is no standardized way of assessing appetitive behaviors or parents' perceptions of their children's appetite. Greater understanding of these factors could improve clinical guidance regarding parent feeding behaviors. We therefore aimed to assess parent perceptions of child weight, and parent reports of child appetite using the Baby Eating Behavior Questionnai...
Source: Appetite - April 15, 2024 Category: Nutrition Authors: Sarah Ann Duck Elena Jansen Afroditi Papantoni Aerial Sheltry Daphne Koinis-Mitchell Viren D'Sa Sean Deoni Timothy H Moran Robert L Findling Peter J Mogayzel Susan Carnell Source Type: research
Inquilinus limosus, an atypical Gram-negative rod from lung transplant recipients and cystic fibrosis patients
Ann Biol Clin (Paris). 2024 Apr 1;82(2):1-2. doi: 10.1684/abc.2024.1876. Online ahead of print.NO ABSTRACTPMID:38616576 | DOI:10.1684/abc.2024.1876 (Source: Annales de Biologie Clinique)
Source: Annales de Biologie Clinique - April 15, 2024 Category: Biochemistry Authors: Eric Farfour St éphane Corvec Thomas Guillard H élène Revillet Source Type: research
Parental perceptions of body weight and appetite in infants and toddlers with cystic fibrosis
Appetite. 2024 Apr 13:107357. doi: 10.1016/j.appet.2024.107357. Online ahead of print.ABSTRACTNutritional status has clinical relevance and is a target of guidance to parents of children with cystic fibrosis (CF). Growth is routinely monitored in CF clinics but there is no standardized way of assessing appetitive behaviors or parents' perceptions of their children's appetite. Greater understanding of these factors could improve clinical guidance regarding parent feeding behaviors. We therefore aimed to assess parent perceptions of child weight, and parent reports of child appetite using the Baby Eating Behavior Questionnai...
Source: Appetite - April 15, 2024 Category: Nutrition Authors: Sarah Ann Duck Elena Jansen Afroditi Papantoni Aerial Sheltry Daphne Koinis-Mitchell Viren D'Sa Sean Deoni Timothy H Moran Robert L Findling Peter J Mogayzel Susan Carnell Source Type: research
Inquilinus limosus, an atypical Gram-negative rod from lung transplant recipients and cystic fibrosis patients
Ann Biol Clin (Paris). 2024 Apr 1;82(2):1-2. doi: 10.1684/abc.2024.1876. Online ahead of print.NO ABSTRACTPMID:38616576 | DOI:10.1684/abc.2024.1876 (Source: Annales de Biologie Clinique)
Source: Annales de Biologie Clinique - April 15, 2024 Category: Biochemistry Authors: Eric Farfour St éphane Corvec Thomas Guillard H élène Revillet Source Type: research
Inquilinus limosus, an atypical Gram-negative rod from lung transplant recipients and cystic fibrosis patients
Ann Biol Clin (Paris). 2024 Apr 1;82(2):1-2. doi: 10.1684/abc.2024.1876. Online ahead of print.NO ABSTRACTPMID:38616576 | DOI:10.1684/abc.2024.1876 (Source: Annales de Biologie Clinique)
Source: Annales de Biologie Clinique - April 15, 2024 Category: Biochemistry Authors: Eric Farfour St éphane Corvec Thomas Guillard H élène Revillet Source Type: research
The effect of respiratory muscle training on children and adolescents with cystic fibrosis: a systematic review and meta-analysis
Cystic fibrosis is a chronic genetic disease that can affect the function of the respiratory system. Previous reviews of the effects of respiratory muscle training in people with cystic fibrosis are uncertain ... (Source: BMC Pediatrics)
Source: BMC Pediatrics - April 15, 2024 Category: Pediatrics Authors: WenQian Cai, Meng Li, Yi Xu, Mei Li, JiaNan Wang, YaHui Zuo and JinJin Cao Tags: Research Source Type: research
Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with Cystic Fibrosis following liver transplantation: A systematic review
Cystic fibrosis (CF) is a multisystem disease caused by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and resulting in deficient or dysfunctional CFTR protein [1]. F508del is the most prevalent disease-causing variant in people with CF (pwCF), occurring in 80 –85 % of cases [2,3]. The prognosis has largely been determined by the pulmonary complications of the disease. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 13, 2024 Category: Respiratory Medicine Authors: Ilaria Testa, Giuseppe Indolfi, Rossa Brugha, Henkjan J Verkade, Vito Terlizzi Tags: Review Source Type: research
The prevalence of developmental defects of enamel in people with cystic fibrosis: a systematic review
Oral health impacts systemic health, individual well-being, and quality of life. It is important to identify conditions that may exacerbate oral disease to aid public health and policy development and promote ... (Source: BMC Oral Health)
Source: BMC Oral Health - April 12, 2024 Category: Dentistry Authors: Fiona O ’Leary, Niamh Coffey, Martina Hayes, Francis Burke, Mairéad Harding and Barry Plant Tags: Research Source Type: research
The evolving focus of cystic fibrosis microbiome research
For more than two decades, culture-independent microbiome analysis has been employed in efforts to improve clinical outcomes for those with CF and to gain a better fundamental understanding of polymicrobial infections. The use of these techniques began during the 1990s [1,2], in response, at least in part, to growing concerns over the emergence of new infectious syndromes. Pathogens such as Burkholderia cepacia [3] and rapidly growing nontuberculous mycobacteria [4] were increasingly associated with sudden and profound deterioration in lung function in persons with CF (PwCF) and the potential for other “novel” species ...
Source: Journal of Cystic Fibrosis - April 10, 2024 Category: Respiratory Medicine Authors: Steven L. Taylor, Geraint B. Rogers Source Type: research
Chronic rhinosinusitis in people with CF, a rapidly changing field
Chronic rhinosinusitis (CRS) has been for long addressed as a hallmark of cystic fibrosis (CF). It has been reported to affect the majority of people with CF (pwCF), with almost 100 % of patients revealing pathologic findings in CT scans, which are currently replaced by MRI scans to reduce exposure to ionising radiation and to enhance differentiation of soft tissue structures [1,2]. The burden of sinonasal symptoms markedly impairs a patient ´s well-being, and indeed, to a greater extent than “some rhinitis” is generally expected to bother general health. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 10, 2024 Category: Respiratory Medicine Authors: Jochen G. Mainz, Franziska Duckstein, Carlos Zagoya, Assen Koitschev Source Type: research
Azithromycin reduces bronchial wall thickening in infants with cystic fibrosis
The efficacy of azithromycin as an anti-inflammatory agent in infants with CF was investigated in the COMBAT-CF study [1]. Children with CF diagnosed by newborn screening were 1:1 randomly assigned to maintenance treatment with azithromycin or placebo from diagnosis until age of three, stratified by site. The primary outcome measure was structural airway disease as assessed on chest computed tomography scans (CTs) by the manual Perth-Rotterdam Annotated Grid Morphometric for CF (PRAGMA-CF) scoring method [2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 6, 2024 Category: Respiratory Medicine Authors: Yuxin Chen, Jean-Paul Charbonnier, Eleni-Rosalina Andrinopoulou, Peter D. Sly, Stephen M. Stick, Harm A.W.M. Tiddens, COMBAT-CF Study Group Source Type: research
