Fix FoxG1 deletion in my son using CRISPR

Question

[vivek.foxg1]

Hi My three year old son was diagnosed with FoxG1 deletion that is affecting him terribly (unable to sit walk talk eat, etc. I believe that with CRISPR if the FoxG1 Gene is edited / replaced, my son Kush should be cured. The question I have is how does the FoxG1 gene get created (synthesised ) in the first place.

The way I understand CRISPR will work is below, The Crisper CAS 9 will have the target corrected DNA / RNA for the FoxG1 gene attached to it a s a transport, once in the cell it will go and look for the Broken FoxG1 material , cut it out and replace that with the corrected FoxG1 RNA code.

Several questions here, 1. How long / big is the FoxG1 Gene RNA. How many base pairs long. 2. Is it possible to edit cut / paste the full FoxG1 to get rid of the defective code and replace with the correct code. 3. Another question is that the FoxG1 code that will be sent as a payload with the CRISPR CAS9 system, where do you get that from in the first place. Is this a synthesized material made using nanotechnology is this organic matter from donor cells from somewhere.

Excuse my ignorance if. I am am parent that wants their child fixed and i can raise money for this. does anyone want a grant to start working on this . I am all ears.

Please feel free to contact at anytime of day or night. TOGETHER WE WILL FIND A CURE

[Zev.Kronenberg]

Are you associated with: https://foxg1.com ?

[Sukhi Singh]

Another specific forum(better) to ask this would be http://biology.stackexchange.com/. Hope you get all the information you want.

Answer 1

[jotan]

Dear Vivek,

Unfortunately CRISPR is in very early stages of development and it will be many years before this technology can be used for therapy.

In addition, since the FOXG1 gene is important for early development, it is very very unlikely that correcting the gene would be able to reverse this. Be extremely cautious if anyone promises otherwise.

The best thing to do is to make the most of existing therapies.

As a side note, I would also suggest removing your phone number from this post.

Good luck!

Answer 2

[chen]

Hi vivek,

I feel sorry about your son and understand your wish, but currently CRISPR is only able to edit DNA of embryos, it's not so powerful to edit all cells of one (~ 50,000,000,000,000 cells) . I hope one day scientists will achieve that, but it's not easy.

For your question, FoxG1 is on Chromosome 14, about 3205 bp long. Yes, it is a very small gene. You can get more information about FoxG1 from https://www.ncbi.nlm.nih.gov/gene/2290

[dario.garvan]

It doesn't need to be present in every living cell but only the cell types which it is naturally expressed in.

Answer 3

[Zev.Kronenberg]

vivek.foxg1,

Below you will find two resources that may help answer your questions. The first link will tell you more about the FoxG1 gene and the second link is a well told story about CRISPR.

Here is a link to a human genome browser for FoxG1.

UCSC genome browser

Here is a very nice podcast that discusses what CRISPR is:

CRISPR radio lab

Answer 4

[alons]

Hi Vivek,

So sorry to hear about your son.

I advise you to contact Editas Medicine company which was founded by Jeniffer A. Doudna and Feng Zhang. To the best of my knowledge they are the pioneers of the technology in human genomic editing. Their aim is to provide the clinic with genome editing via Crispr CAS9 technology. Their website: http://www.editasmedicine.com/

In theory, this is possible in early developmental stages and tissue specific regions, all the sequences and tools are generated synthetically and it's work in progress.

Wish you the best of luck!