Gene Therapy And Cystic Fibrosis

Topics:	Gene
Words:	452
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There are many different types of viral vectors that are used to deliver a corrected CFTR gene. The Adenoviral vector was one of the first vectors to be tested to see if it couls successfully deliver the corrected gene. Ad vectors don’t become a part of the host genome and remain episomal which means they can replicate independently. Just a few years after replicating the CFTR gene the first trial was carried out using this vector. Zabner et al. gave the Ad-CFTR to 3 CF patients. This was done by administering the Ad-CFTR to the nasal epithelium because it has a similar cell composition to lower airways. A conclusion about efficacy was not reached because of the lower number of patients in the trial but 9 more trials were carried out between 1993 and 2001. These trails showed many promising results; in the nasal epithelium of some patients there was partial correction of chloride transport and in some subjects low level gene transfer was accomplished. But lung inflammation was seen to be a side effect but depended on the dosage amount so could be controlled.

Another viral vector being assessed is the Adeno-Associated vector. Like the Ad vector this does not integrate into the genome and stays largely episomal once inside the nucleus. May serotypes have been recognised and protein shells from serotypes 1, 5, 6, 8 and 9 seem to be most effective in transducing airway epithelial cells. Six clinical studies were carried out between 1999 and 2007 led by Targeted Genetics Corp. patients were firstly given a single dose in phase 1 where the AVV serotype 2 vector was administered to the nose, sinuses and lungs. This showed administration was safe but didn’t provide an opportunity assess how efficient the vector was at CFTR expression. One hundred subjects were given a repeat dosage, but this did not improve lung function because of the development of an anti-viral immune response.

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Cytoplasmic viruses can also be used as vectors to deliver the corrected gene. Numerous cytoplasmic RNA viruses have been identified for airway gene transfer. The murine parainfluenza virus type 1 (SeV), the human respiratory syncytial virus and the human parainfluenza virus have all be proven capable to infect AEC’s through the apical membrane. All of the above viruses have a negative strand RNA genome. They also can replicate in the cytoplasm unlike other viral vectors. SeV has been tested in animal models but recurring administration wasn’t achievable due to the growth of counteracting antibodies against the vector so as a result did not proceed into clinical trials.

Lentivirial vectors contrast to the others as they do integrate into the host genome. These are regularly pseudotyped with an envelope of glycoprotein resulting in the ability to infect many different types of tissues.

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Gene Therapy And Cystic Fibrosis. (2022, February 17). Edubirdie. Retrieved April 19, 2024, from https://edubirdie.com/examples/gene-therapy-and-cystic-fibrosis/

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Gene Therapy And Cystic Fibrosis [Internet]. Edubirdie. 2022 Feb 17 [cited 2024 Apr 19]. Available from: https://edubirdie.com/examples/gene-therapy-and-cystic-fibrosis/

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