Gene therapy for cystic fibrosis.

Coutelle, Charles; Caplen, Natasha J.; Hart, SL; Huxley, Clare; Williamson, Robert

doi:10.1136/adc.68.4.437

Cited by 24 publications

(4 citation statements)

References 33 publications

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“…1,2 The mean age at death for CF is 29 years; the most common cause of death is respiratory failure. 3 Although the cellular defect in CF has been characterized for more than a decade, 4 and therapeutic advances have been substantial, [5][6][7][8][9] CF remains uniformly fatal.…”

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confidence: 99%

End-of-Life Care in Cystic Fibrosis

et al. 1997

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The model of comfort care developed in childhood cancer does not adequately describe the combination of preventive, therapeutic, and palliative care given at the end of life for CF at our institution. The majority of CF patients continued to receive intravenous antibiotics and/or oral vitamin preparations while being treated with opiates for terminal pain and dyspnea. Small doses of opiates seem to be effective in the treatment of the pain and dyspnea at the end of life in CF.

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confidence: 99%

End-of-Life Care in Cystic Fibrosis

et al. 1997

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“…Liposomes have been successfully used to deliver a CFTR expression plasmid to airway epithelia of transgenic mice [28], and the first trial with this type of vector has started in the United Kingdom. In the meantime, search for other gene delivery systems such as mammalian or yeast artificial chromosomes and cell targeting using, for instance, cell surface receptor ligands [12] will continue.…”

Section: Current Status Of Newer Therapeutic Approachesmentioning

confidence: 99%

New perspectives in understanding and management of the respiratory disease in cystic fibrosis

Suter

1994

Eur J Pediatr

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In the past 40 years, the mean survival of patients with cystic fibrosis (CF) has increased from less than 1 year to 30 years. The identification of the gene mutated in CF in 1989 has already been followed by the first phase of somatic gene therapy in 1993. The target organ of somatic gene therapy is the respiratory epithelium, which is progressively damaged by the chronic infection and inflammation characteristic of the disease. Since in the future, more patients may benefit from somatic gene therapy, the understanding of the mechanisms leading to chronic infection and inflammation becomes increasingly important. In the future, current therapeutic measures to protect the respiratory epithelium from damage, such as intravenous antimicrobial treatment, will be improved by the additional delivery of new drugs to the bronchial tree by aerosol. Amiloride and recombinant human DNAse administered by this route have the potential to improve mucociliary clearance. Antibiotics as well as protease inhibitors delivered by aerosol should contribute to prevent damage by infection and inflammation in order to increase the probability of successful somatic gene therapy in this disease.

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“…There was no activation of complement, although it is thought that free poly-L-lysine can activate complement by the alternative pathway. Airway epithelial cells represent a good target for gene delivery as transfection efficiencies in the region of 10% may be enough to relieve the symptoms cause by cystic fibrosis disease [79].…”

Section: Gene Delivery To Epithelial Cellsmentioning

confidence: 99%

Ligand-targeted receptor-mediated vectors for gene delivery

Deonarain

1998

Expert Opinion on Therapeutic Patents

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Gene therapy promises to cure human genetic diseases. One of the main obstacles to fulfilling this promise is in the ability to target a gene to a significant population of cells and express it at adequate levels for a long enough period of time. Viral methods for gene delivery have been studied for a number of years and are effective vectors for gene transfer. The great majority of gene therapy clinical trials currently in progress use retroviruses or adenoviruses. However, there are concerns for their clinical use because of possible risks of mutagenesis, immunogenic side-effects and toxicity. In addition to this, there are other limitations, including the size of gene that can be transferred. Over the last ten years, a new approach has emerged that has increasingly gathered speed thanks to advances in receptor cell biology and antibody production. This method involves ligand-targeted receptor mediated endocytosis (RME) of 'polyplexes'. Here, synthetic complexes are composed of a cell-specific targeting ligand, coupled to a DNA binding element and endosmolytic function. These complexes are able to deliver genes to cells in a receptor-specific manner, without any viral DNA sequences or packaging constraints. There are now many ligand/receptor systems under investigation, each one demonstrating successful gene transfer with a higher level of tissue specificity than viruses can offer. This review describes most of these systems and looks ahead to an era where cellspecific gene delivery may be a main stream gene therapy, treatment modality.

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Gene therapy for cystic fibrosis.

Cited by 24 publications

References 33 publications

End-of-Life Care in Cystic Fibrosis

End-of-Life Care in Cystic Fibrosis

New perspectives in understanding and management of the respiratory disease in cystic fibrosis

Ligand-targeted receptor-mediated vectors for gene delivery

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