Genetic diseases

Can anyone explain the following in simple English please:

- How do they identify which gene does what?
- How do they know which gene causes which disease?
- How do they turn the gene off so that the disease is stopped?

Cheers.

Originally posted by shavixmir
Can anyone explain the following in simple English please:

- How do they identify which gene does what?
- How do they know which gene causes which disease?
- How do they turn the gene off so that the disease is stopped?

Cheers.

Good Questions!

To begin with the last one:

Gene therapy is still at its most intiail stages. Currently, there is no way to fix a gene that is available for therapy. However, there are attempts of introducing the "correct" form of the gene, using viruses and other methods. Deactivation of genes is also possible, and is done in labs in the form of RNA interference, but the procedure is still not ready to be used as a therapy.

Now, to answer your other questions.
- How do they identify which gene does what?
- How do they know which gene causes which disease?

Well, this two questions are very similar, as the role of the gene is related to the disease caused when the gene is defective.

Before I continue, we have to acknowledge two concepts:

Genotype: The genes you have. For example, someone can have a gene for O blood type from his father and a gene for B blood type from his mother (or visa verse, it matters not).

Phenotype: The observed result of the genotype ; In our example a B blood type - the O gene does not effect the phenotype in this example.


The "conservative" approach is using two methods: Forward Genetics and Reverse Genetics.

Forward Genetics is seeing a phenotype (in our case, a genetic disease), and comparing the genes of ones so afflicted with those of healthy individuals.

Reverse Genetics is causing a mutation in a specific gene, to see how it changes the phenotype - of course, you don't do it with human beings! you can, however, do it to human cell lines.


The "Genomics Age" methods are based on comparing known genes to the gene you want to research , in order to asses what it does ; I haven't heard about any genetic diseases that were found this way (then again, that's just me) , but it is a very useful approach in learning about genes functions and interactions.


Of course, this is merely the tip of the iceberg but I hope it helps.

Originally posted by Retrovirus
Good Questions!

To begin with the last one:

Gene therapy is still at its most intiail stages. Currently, there is no way to fix a gene that is available for therapy. However, there are attempts of introducing the "correct" form of the gene, using viruses and other methods. Deactivation of genes is also possible, and is done in labs in the form of RN ...[text shortened]... nteractions.


Of course, this is merely the tip of the iceberg but I hope it helps.

Yes. Thank you!

Is it okay if I PM you with various questions?

Originally posted by shavixmir
Yes. Thank you!

Is it okay if I PM you with various questions?

If that's your picture, I can see why you would be interested in gene therapy🙂

Originally posted by shavixmir
Yes. Thank you!

Is it okay if I PM you with various questions?

Why don't post them here?

Originally posted by sonhouse
If that's your picture, I can see why you would be interested in gene therapy🙂

Thank you.