Genetic medicines for CF: Hype versus reality

Genetic medicines for CF: Hype versus reality

Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of "genetic medicines" including mRNA delivery, as well as genome editing and mRNA repair-based strategies....

Disgrifiad llawn

Manylion Llyfryddiaeth
Prif Awduron:	Alton, E, Boyd, A, Davies, J, Gill, D, Griesenbach, U, Harrison, P, Henig, N, Higgins, T, Hyde, S, Innes, J, Korman, M
Fformat:	Journal article
Iaith:	English
Cyhoeddwyd:	Wiley 2016

Eitemau Tebyg

EVALUATION OF SAFETY AND GENE EXPRESSION WITH A SINGLE DOSE OF PGM169/GL67A ADMINISTERED TO THE NOSE AND LUNG OF INDIVIDUALS WITH CF: THE UK CF GENE THERAPY CONSORTIUM "PILOT STUDY"
gan: Davies, J, et al.
Cyhoeddwyd: (2009)

Update on the UK CF Gene Therapy Consortium Multidoes, non-viral, gene therapy trial
gan: Alton, E, et al.
Cyhoeddwyd: (2012)

SAFETY AND EXPRESSION OF A SINGLE DOSE OF LIPID-MEDIATED CFTR GENE THERAPY TO THE UPPER AND LOWER AIRWAYS OF PATIENTS WITH CF
gan: Davies, J, et al.
Cyhoeddwyd: (2011)

UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL
gan: Alton, E, et al.
Cyhoeddwyd: (2012)

UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL
gan: Alton, E, et al.
Cyhoeddwyd: (2012)

A PHASE 2B DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF NON-VIRAL MEDIATED GENE THERAPY FOR CF
gan: Alton, E, et al.
Cyhoeddwyd: (2013)

EVALUATION OF SAFETY AND GENE EXPRESSION WITH A SINGLE DOSE OF PGM169/GL67A ADMINISTERED TO THE NOSE AND LUNG OF INDIVIDUALS WITH CYSTIC FIBROSIS: THE UK CF GENE THERAPY CONSORTIUM "PILOT STUDY''
gan: Davies, J, et al.
Cyhoeddwyd: (2009)

TOXICOLOGY STUDIES IN SUPPORT OF THE UK CF GENE THERAPY CONSORTIUM'S MULTI-DOSE CLINICAL TRIAL
gan: Griesenbach, U, et al.
Cyhoeddwyd: (2011)

Production of SIV-F/HN: a new Lentivirus vector for CF gene therapy
gan: Hyde, S, et al.
Cyhoeddwyd: (2015)

UKCF gene therapy consortium tracking study: Sputum and serum calprotectin are useful novel measurements in CF exacerbation
gan: Gray, R, et al.
Cyhoeddwyd: (2007)

SAFETY AND EXPRESSION OF A SINGLE DOSE OF LIPID-MEDIATED CFTR GENE THERAPY TO THE UPPER AND LOWER AIRWAYS OF PATIENTS WITH CYSTIC FIBROSIS
gan: Davies, G, et al.
Cyhoeddwyd: (2011)

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
gan: Alton, E, et al.
Cyhoeddwyd: (2013)

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis.
gan: Alton, E, et al.
Cyhoeddwyd: (2013)

EXERCISE CAPACITY AND PHYSICAL ACTIVITY IN PATIENTS WITH CF: DATA FROM THE UK CF GENE THERAPY CONSORTIUM (UKCFGTC) 'RUN-IN' STUDY
gan: Saunders, C, et al.
Cyhoeddwyd: (2010)

LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS; THE RUN-IN STUDY
gan: Alton, E, et al.
Cyhoeddwyd: (2010)

From hype to reality: data science enabling personalized medicine
gan: Holger Fröhlich, et al.
Cyhoeddwyd: (2018-08-01)

Three‐Dimensional Bioprinting in Regenerative Medicine: Reality, Hype, and Future
gan: Anthony Atala, et al.
Cyhoeddwyd: (2019-08-01)

DEVELOPMENT, PRODUCTION AND EVALUATION OF CLINICAL GRADE CFTR EXPRESSION PLASMID FOR CF LUNG GENE THERAPY
gan: Gill, DR, et al.
Cyhoeddwyd: (2010)

LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS: THE RUN-IN STUDY
gan: Alton, E, et al.
Cyhoeddwyd: (2010)

Platform switching: Hype or reality?
gan: Shefali Singla, et al.
Cyhoeddwyd: (2015-01-01)

E-Learning: The Hype and the Reality
gan: Gráinne Conole
Cyhoeddwyd: (2004-09-01)

Self-reactive CFTR T cells in humans: implications for gene therapy.
gan: Calcedo, R, et al.
Cyhoeddwyd: (2013)

Pre-existing immunity to human parainfluenza virus (hPIV) does not affect rSIV.F/HN-mediated transduction efficiency
gan: Pytel, K, et al.
Cyhoeddwyd: (2016)

Moving lentiviral-based gene therapy into a first-in-man CF trial
gan: Griesenbach, D, et al.
Cyhoeddwyd: (2015)

Delivery of genes into the CF airway
gan: Gill, D, et al.
Cyhoeddwyd: (2014)

Personalised medicine: the promise, the hype and the pitfalls
gan: Feiler, T, et al.
Cyhoeddwyd: (2017)

The Promise and the hype of 'personalised medicine'
gan: Maughan, T
Cyhoeddwyd: (2017)

Personalized medicine: hope or hype?
gan: Salari, K, et al.
Cyhoeddwyd: (2012)

Towards a first-in-human trial with a pseudotyped lentivirus
gan: Alton, EW, et al.
Cyhoeddwyd: (2020)

Neurophobia amongst medical students: Hype or reality
gan: Suzanne Murphy, et al.
Cyhoeddwyd: (2024-01-01)

Biotics in pediatrics: Separating hype from reality
gan: Hania Szajewska
Cyhoeddwyd: (2024-03-01)

Prognostic role of inflammatory markers: Hype or reality?
gan: Richa Vaish, et al.
Cyhoeddwyd: (2020-01-01)

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.
gan: Alton, E, et al.
Cyhoeddwyd: (2014)

Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung
gan: Alton, E, et al.
Cyhoeddwyd: (2012)

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung
gan: Alton, E, et al.
Cyhoeddwyd: (2014)

REPEAT ADMINISTRATION OF GL67A/PGM169 IS FEASIBLE, SAFE, AND PRODUCES ENDOGENOUS LEVELS OF CFTR EXPRESSION AFTER 12 DOSES
gan: Alton, E, et al.
Cyhoeddwyd: (2012)

Gene therapy for respiratory diseases; progress and a changing context
gan: Alton, EWFW, et al.
Cyhoeddwyd: (2020)

UK Shale Gas – Hype, Reality and Difficult Questions
gan: Rogers, H
Cyhoeddwyd: (2013)

Beyond the Hype: Unveiling the Realities of Crypto Trading Journeys
gan: Muhammad Sohail, et al.
Cyhoeddwyd: (2023-12-01)

Bridging between hype and implementation in medical extended reality
gan: Oscar Freyer, et al.
Cyhoeddwyd: (2023-12-01)