S
splog23
Guest
Hey,
I am a complete newcomer to this forum, and probably have no idea what I am talking about. DON'T JUDGE!!!
I think I have come up with a possible theory for Gene Therapy for Cystic Fibrosis.
This may be an already known/unavailable theory but I haven't found anything like it so far.
Please, if you can see I have made a mistake in this idea do not hesitate to tell me.
I am probably going to make a mistake as although I understand the basics of C.F and gene therapy, I do not know enough to be certain that this would work in theory.
I was researching vectors in gene therapy and after searching many pages for info about the vectors, i came across some of the problems with viral vectors. Mainly, the fact that:
Retroviruses, when infected into a host cell, could randomly insert the genetic material into a chromosome and cause uncontrolled cell division (ie cancer) and to date 5 children have developed leukaemia as a result of this.
Adenoviruses and Adeno-associated viruses, while being safe in terms of 'chromosome placement', have a limited cloning capacity and so large genes are not suitable for use in a AAV (Adeno-associated virus). It is also very difficult to produce.
Viral vectors are problematic as the immune response will detect these as threats and percieve them as invading pathogens. Thus, the virus/ new genetic information will be destroyed and no effect will take place.
I came up with the idea of perhaps using a nanovector. This would be a method of transporting the genetic material in a fullerene-based nanostructure. this nanovector would in theory be a 'cage' which holds an un-mutated CFTR gene.
The nanovector would of course have to be small enough to be easily injected into a cell but large enough to house the 230000 base pairs that make up the CFTR gene, however this is most definitely possible with our understanding of nanotechnology.
Anyway, this nanovector, which I shall call 'X', would be injected into the somatic cell, passing through the membrane of the cell. As it is not viral, the immune response will be minimal as 'X' would not be detected as a threat. 'X' would then travel directly to the nucleus and through endocytosis (being absorbed by the cell) would be able to enter the nucleus. In the nucleus 'X' would break up when it had arrived at the correct site on the chromosome This is because the CFTR gene has a unique 'R' domain that other ABC transporters do not. As a result of this 'X' would be able to single out the R domain and would break up, releasing the genetic material into the correct area in the chromosome.
I am not sure if a replicating gene would be possible if placed inside a nanovector but if it is, assuming I haven't made a fatal error, this method of gene therapy would be able to replace the faulty CFTR gene that causes Cystic Fibrosis.
Please, please, please respond to me telling em what is correct or wrong in this thread. I am simply trying to help as many people as i can but i will need your help to ensure this.
Thanks!
I am a complete newcomer to this forum, and probably have no idea what I am talking about. DON'T JUDGE!!!
I think I have come up with a possible theory for Gene Therapy for Cystic Fibrosis.
This may be an already known/unavailable theory but I haven't found anything like it so far.
Please, if you can see I have made a mistake in this idea do not hesitate to tell me.
I am probably going to make a mistake as although I understand the basics of C.F and gene therapy, I do not know enough to be certain that this would work in theory.
I was researching vectors in gene therapy and after searching many pages for info about the vectors, i came across some of the problems with viral vectors. Mainly, the fact that:
Retroviruses, when infected into a host cell, could randomly insert the genetic material into a chromosome and cause uncontrolled cell division (ie cancer) and to date 5 children have developed leukaemia as a result of this.
Adenoviruses and Adeno-associated viruses, while being safe in terms of 'chromosome placement', have a limited cloning capacity and so large genes are not suitable for use in a AAV (Adeno-associated virus). It is also very difficult to produce.
Viral vectors are problematic as the immune response will detect these as threats and percieve them as invading pathogens. Thus, the virus/ new genetic information will be destroyed and no effect will take place.
I came up with the idea of perhaps using a nanovector. This would be a method of transporting the genetic material in a fullerene-based nanostructure. this nanovector would in theory be a 'cage' which holds an un-mutated CFTR gene.
The nanovector would of course have to be small enough to be easily injected into a cell but large enough to house the 230000 base pairs that make up the CFTR gene, however this is most definitely possible with our understanding of nanotechnology.
Anyway, this nanovector, which I shall call 'X', would be injected into the somatic cell, passing through the membrane of the cell. As it is not viral, the immune response will be minimal as 'X' would not be detected as a threat. 'X' would then travel directly to the nucleus and through endocytosis (being absorbed by the cell) would be able to enter the nucleus. In the nucleus 'X' would break up when it had arrived at the correct site on the chromosome This is because the CFTR gene has a unique 'R' domain that other ABC transporters do not. As a result of this 'X' would be able to single out the R domain and would break up, releasing the genetic material into the correct area in the chromosome.
I am not sure if a replicating gene would be possible if placed inside a nanovector but if it is, assuming I haven't made a fatal error, this method of gene therapy would be able to replace the faulty CFTR gene that causes Cystic Fibrosis.
Please, please, please respond to me telling em what is correct or wrong in this thread. I am simply trying to help as many people as i can but i will need your help to ensure this.
Thanks!