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toggle navigation news about us about about about who are we consortium timeline profiles of the consortium strategy group frequently asked questions all trials registered initiative contact details about this website mailform videos funding funding funding funding funding overview flutterby fundraisers the cystic fibrosis trust just gene therapy cystic fibrosis cf cf cf what is cystic fibrosis? history of cystic fibrosis discovery of the cftr gene what causes cystic fibrosis? cftr protein structure gene therapy introduction to gene therapy why use gene therapy to treat cf? other cf gene therapy groups successful applications of gene therapy cf gene therapy clinical trials clinical programme clinical clinical clinical gl67a pgm169 - our first clinical trial product the tracking study the run in study single dose clinical trial (2009-2011) multi dose clinical trial (2012-2014) multi dose clinical trial protocol public meeting, 30 may 2015 research nonviral vector development aerosol delivery of gene transfer vectors assay development lentiviral vector development lentiviral production publications journal articles conference presentations book chapters public presentations blog flutterby fundraisers to sponsor consortium scientist 8 months ago new publication - gelinas et al 2017 - media supplements to improve lentiviral vector production yields 10 months ago gene therapy funding update 10 months ago consortium student live facebook interview from asgct 1 year ago welcome to the uk cfgtc website. × close internet explorer users: we simply don't have the resources to make this site work better on ie (we don't have any modern pcs). we recommend chrome . gene therapy funding update the ukcfgtc is pleased to announce that we have received £2.7m to undertake a phase 1/2a nose trial in cf patients using our wave 2 product, delivering the cftr gene using a novel lentivirus . this latest support, which builds on many years of gene therapy funding from the cystic fibrosis trust , the national institute for health research (nihr) and the medical research council (mrc), has been awarded by the wellcome trust/department of health’s health innovation challenge fund ( hicf ). at the same time the cystic fibrosis trust have awarded an additional £0.5m to continue to support the scientific work underpinning this latest trial over the next two years. we aim to recruit 24 patients into the phase 1/2a nose trial which will last around 9 months. the study will assess safety, and any changes in molecular endpoints, to provide evidence for the efficacy of the lentivirus. the start point of the trial will depend on the time required for manufacture of the wave 2 product for clinical delivery; we will further update on timelines once these manufacturing data are available. we are now focusing our research and development efforts on wave 2, which has proved to be considerably more efficient than the wave 1 product (delivering the cftr gene via liposomes). however, the latter, which led to a stabilisation of lung function significantly different to the decline seen in a placebo group, continues to be discussed with potential commercial partners. we will update further on the outcome of these discussions as soon as possible. 10 months ago consortium gene therapy clinical trial results published the consortium are pleased to announce the publication of the results from our multi dose gene therapy clinical trial in lancet respiratory medicine . one hundred and thirty six patients aged 12 and above were randomly assigned to either 5ml of nebulised pgm169/gl67a (gene therapy) or saline (placebo) at monthly intervals over 1 year. lung function was evaluated using a common clinical measure fev1. the clinical trial reached its primary endpoint with patients who received therapy having a significant, if modest benefit in lung function compared with those receiving a placebo. after a year of treatment, in the 62 patients who received the gene therapy, fev1 was 3.7% greater compared to placebo. the trial is the first ever to show that repeated doses of a gene therapy can have a meaningful effect on the disease and change the lung function of patients. more details here . 3 years ago latest consortium publications multiple inhibitory factors act in the late phase of hiv-1 replication: a systematic review of the literature gelinas jf et al. microbiology and molecular biology reviews (2018) 6 months ago assessment of selected media supplements to improve f/hn lentiviral vector production yields. gelinas jf et al. scientific reports (2017) 10 months ago preparation for a first-in-man lentivirus trial in patients with cystic fibrosis alton ew et al. thorax (2016) 1 year ago all publications about us the consortium groups are based in three of the leading uk universities. prior to the formation of the consortium each group had conducted at least one clinical trial for gene therapy. therefore, our researchers have been at the forefront of this field for over 20 years. learn more research our research is focussed on the development of gene therapies for cf. taking research ideas from the lab to the clinic is beyond the reach of most academic groups. the consortium is different as we have aligned our different groups to focus on their key talents to ensure that we continue to progress. learn more publications since 2004 ukcfgtc members have published over 100 papers in peer reviewed journals and have presented many posters and presentations at international conferences. where possible we will provide pdfs of publications. however, most of our conference posters and presentations are available. learn more ukcfgtc , university of oxford , imperial college london & university of edinburgh tweet

URL analysis for cfgenetherapy.org.uk

http://www.cfgenetherapy.org.uk/aboutus/article/consortium_timeline
http://www.cfgenetherapy.org.uk/clinical/article/multi_dose_clinical_trial_20122014
http://www.cfgenetherapy.org.uk/clinical/article/the_tracking_study
http://www.cfgenetherapy.org.uk/cysticfibrosis/article/what_causes_cystic_fibrosis
http://www.cfgenetherapy.org.uk/funding/index.php
http://www.cfgenetherapy.org.uk/clinical/index.php
http://www.cfgenetherapy.org.uk/nonpublic/index.html
http://www.cfgenetherapy.org.uk/research/article/nonviral_vector_development
http://www.cfgenetherapy.org.uk/research/article/aerosol_delivery_of_gene_transfer_vectors
http://www.cfgenetherapy.org.uk/research/index.php
http://www.cfgenetherapy.org.uk/genetherapy/article/why_use_gene_therapy_to_treat_cf
http://www.cfgenetherapy.org.uk/funding/article/funding_overview
http://www.cfgenetherapy.org.uk/news/article/cystic_fibrosis_gene_therapy_clinical_trial_demonstrates_beneficial_effect_on_lung_function
http://www.cfgenetherapy.org.uk/aboutus/article/about_this_website
http://www.cfgenetherapy.org.uk/aboutus/article/profiles_of_the_consortium_strategy_group
hicfund.org.uk
mrc.ac.uk
cysticfibrosis.org.uk
ed.ac.uk
ox.ac.uk
nihr.ac.uk
ic.ac.uk

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