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....

Бүрэн тодорхойлолт

Номзүйн дэлгэрэнгүй
Үндсэн зохиолчид:	Alton, E, Boyd, A, Davies, J, Gill, D, Griesenbach, U, Harrison, P, Henig, N, Higgins, T, Hyde, S, Innes, J, Korman, M
Формат:	Journal article
Хэл сонгох:	English
Хэвлэсэн:	Wiley 2016

Ижил төстэй зүйлс

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"
-н: Davies, J, зэрэг
Хэвлэсэн: (2009)

Update on the UK CF Gene Therapy Consortium Multidoes, non-viral, gene therapy trial
-н: Alton, E, зэрэг
Хэвлэсэн: (2012)

SAFETY AND EXPRESSION OF A SINGLE DOSE OF LIPID-MEDIATED CFTR GENE THERAPY TO THE UPPER AND LOWER AIRWAYS OF PATIENTS WITH CF
-н: Davies, J, зэрэг
Хэвлэсэн: (2011)

UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL
-н: Alton, E, зэрэг
Хэвлэсэн: (2012)

UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL
-н: Alton, E, зэрэг
Хэвлэсэн: (2012)

A PHASE 2B DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF NON-VIRAL MEDIATED GENE THERAPY FOR CF
-н: Alton, E, зэрэг
Хэвлэсэн: (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''
-н: Davies, J, зэрэг
Хэвлэсэн: (2009)

TOXICOLOGY STUDIES IN SUPPORT OF THE UK CF GENE THERAPY CONSORTIUM'S MULTI-DOSE CLINICAL TRIAL
-н: Griesenbach, U, зэрэг
Хэвлэсэн: (2011)

Production of SIV-F/HN: a new Lentivirus vector for CF gene therapy
-н: Hyde, S, зэрэг
Хэвлэсэн: (2015)

UKCF gene therapy consortium tracking study: Sputum and serum calprotectin are useful novel measurements in CF exacerbation
-н: Gray, R, зэрэг
Хэвлэсэн: (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
-н: Davies, G, зэрэг
Хэвлэсэн: (2011)

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
-н: Alton, E, зэрэг
Хэвлэсэн: (2013)

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

EXERCISE CAPACITY AND PHYSICAL ACTIVITY IN PATIENTS WITH CF: DATA FROM THE UK CF GENE THERAPY CONSORTIUM (UKCFGTC) 'RUN-IN' STUDY
-н: Saunders, C, зэрэг
Хэвлэсэн: (2010)

LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS; THE RUN-IN STUDY
-н: Alton, E, зэрэг
Хэвлэсэн: (2010)

From hype to reality: data science enabling personalized medicine
-н: Holger Fröhlich, зэрэг
Хэвлэсэн: (2018-08-01)

Three‐Dimensional Bioprinting in Regenerative Medicine: Reality, Hype, and Future
-н: Anthony Atala, зэрэг
Хэвлэсэн: (2019-08-01)

Platform switching: Hype or reality?
-н: Shefali Singla, зэрэг
Хэвлэсэн: (2015-01-01)

DEVELOPMENT, PRODUCTION AND EVALUATION OF CLINICAL GRADE CFTR EXPRESSION PLASMID FOR CF LUNG GENE THERAPY
-н: Gill, DR, зэрэг
Хэвлэсэн: (2010)

LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS: THE RUN-IN STUDY
-н: Alton, E, зэрэг
Хэвлэсэн: (2010)

Self-reactive CFTR T cells in humans: implications for gene therapy.
-н: Calcedo, R, зэрэг
Хэвлэсэн: (2013)

Personalised medicine: the promise, the hype and the pitfalls
-н: Feiler, T, зэрэг
Хэвлэсэн: (2017)

Moving lentiviral-based gene therapy into a first-in-man CF trial
-н: Griesenbach, D, зэрэг
Хэвлэсэн: (2015)

Delivery of genes into the CF airway
-н: Gill, D, зэрэг
Хэвлэсэн: (2014)

Pre-existing immunity to human parainfluenza virus (hPIV) does not affect rSIV.F/HN-mediated transduction efficiency
-н: Pytel, K, зэрэг
Хэвлэсэн: (2016)

The Promise and the hype of 'personalised medicine'
-н: Maughan, T
Хэвлэсэн: (2017)

Personalized medicine: hope or hype?
-н: Salari, K, зэрэг
Хэвлэсэн: (2012)

Neurophobia amongst medical students: Hype or reality
-н: Suzanne Murphy, зэрэг
Хэвлэсэн: (2024-01-01)

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

Prognostic role of inflammatory markers: Hype or reality?
-н: Richa Vaish, зэрэг
Хэвлэсэн: (2020-01-01)

Towards a first-in-human trial with a pseudotyped lentivirus
-н: Alton, EW, зэрэг
Хэвлэсэн: (2020)

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.
-н: Alton, E, зэрэг
Хэвлэсэн: (2014)

Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung
-н: Alton, E, зэрэг
Хэвлэсэн: (2012)

Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung
-н: Alton, E, зэрэг
Хэвлэсэн: (2014)

REPEAT ADMINISTRATION OF GL67A/PGM169 IS FEASIBLE, SAFE, AND PRODUCES ENDOGENOUS LEVELS OF CFTR EXPRESSION AFTER 12 DOSES
-н: Alton, E, зэрэг
Хэвлэсэн: (2012)

Gene therapy for respiratory diseases; progress and a changing context
-н: Alton, EWFW, зэрэг
Хэвлэсэн: (2020)

UK Shale Gas – Hype, Reality and Difficult Questions
-н: Rogers, H
Хэвлэсэн: (2013)

Beyond the Hype: Unveiling the Realities of Crypto Trading Journeys
-н: Muhammad Sohail, зэрэг
Хэвлэсэн: (2023-12-01)

Bridging between hype and implementation in medical extended reality
-н: Oscar Freyer, зэрэг
Хэвлэсэн: (2023-12-01)

Editorial: Personalizing Treatment in IBD: Hype or Reality in 2020?
-н: Fernando Gomollón, зэрэг
Хэвлэсэн: (2021-05-01)