Timeline for Current state of Gene Therapy [closed]
Current License: CC BY-SA 3.0
19 events
| when toggle format | what | by | license | comment | |
|---|---|---|---|---|---|
| May 30, 2016 at 8:43 | history | closed |
rg255 March Ho AliceD♦ kmm WYSIWYG |
Needs more focus | |
| May 29, 2016 at 21:30 | history | edited | David | CC BY-SA 3.0 |
Added missing question mark.
|
| May 29, 2016 at 21:29 | comment | added | David | I've edited the title of the question to include the term, Gene Therapy, which is what your question is about. I have also clarified it. The 43 year-old man may be perhaps someone you know, but isn't relevant here. | |
| May 29, 2016 at 21:25 | answer | added | David | timeline score: 2 | |
| May 29, 2016 at 21:19 | comment | added | Chris♦ | @user137 Please do not use the comments to write an answer. Put these comments together, add a few references and you have an answer. | |
| May 29, 2016 at 20:57 | history | edited | David | CC BY-SA 3.0 |
Clarified the question and gave it a meaningful and relevant title. Added appropriate tag
|
| S May 29, 2016 at 14:42 | history | suggested | another 'Homo sapien' | CC BY-SA 3.0 |
improved formatting
|
| May 29, 2016 at 13:25 | comment | added | another 'Homo sapien' | @user137 yeah man that's too many comments. Putting an answer instead will increase the chances that someone will read all of it ;) | |
| May 29, 2016 at 12:55 | comment | added | Malhar Khushu | @user137 You couldv'e just put this as an answer, I believe you're right! | |
| May 29, 2016 at 11:24 | comment | added | user137 | DNA alongside the CRISPER gene and the guide RNA. The target cells have to get all three components in the right proportions, and they have to choose the good DNA to repair the damaged genomic DNA. Furthermore, you can't simply select correct cells in an in vivo situation, because killing off 90% or more of the cells will kill the patient, so your delivery efficiencies and repair efficiencies must be much much higher than we've achieved in gene delivery so far. | |
| May 29, 2016 at 11:21 | comment | added | user137 | But the biggest barrier to efficient gene editing is probably homology directed repair. When DNA is cut, your cells have 2 ways to fix it, they can just stick the ends together and fill in the gaps. This usually introduces errors and can often simply destroy a gene, and is usually not desired in gene editing, but it's much more efficient. Homology Directed Repair will take a matching copy of the damaged DNA, usually the corresponding gene on the sister chromatid, and use it to copy the sequence into the damaged DNA. This is what allows gene editing to work, but you have to deliver your good | |
| May 29, 2016 at 11:18 | comment | added | user137 | right cell type, and then get that DNA into the nucleus. Once in the nucleus, the DNA can make the protein, in our case a gene editing nuclease. Now the nuclease must find and cut the bad gene in the right way, if using CRISPR, that requires a small guide RNA to target the enzyme to the right place. Depending on how this RNA is delivered, not all cells that get a copy of the CRISPR gene will get the RNA, so these cells will not have their DNA cut. Using ZFN or TALENS avoids the guide RNA requirement, but often have a worse accuracy rate and can cut the DNA at incorrect positions. | |
| May 29, 2016 at 11:15 | comment | added | user137 | and grow as many copies as you need. In a living organism, it becomes much harder. Most of the gene editing nucleases are delivered as a molecule of DNA or mRNA that can then make the enzyme once it gets into a cell. However, delivery of DNA to cells in vivo is an incredible challenge. Modified viruses are more effective than nonviral systems, but have limited carrying capacity and possible immunological effects. Nonviral systems are in still in their infancy after over 20 years of effort. A nonviral gene delivery system must protect DNA from destruction by nucleases, get the DNA into the | |
| May 29, 2016 at 11:11 | comment | added | user137 | What you're interested in is a form of Gene Therapy. This is extremely difficult. There are many strategies currently being researched, and the best strategy to use probably depends on the specific genetic condition. In your question, you mention gene editing, which uses CRISPR/Cas9, Zinc Finger Nucleases, or TALEN endonucleases to cut DNA at a specific site, then relies on the cell's DNA repair mechanism to fix the cut, hopefully with a correct of the gene provided alongside the nuclease enzyme. This is very inefficient in cell culture, but at least in cell culture you can find a good cell | |
| May 29, 2016 at 7:27 | review | Close votes | |||
| May 30, 2016 at 8:45 | |||||
| May 29, 2016 at 5:37 | comment | added | Malhar Khushu | OK, wow, that's a lot of questions. Theoretically, if you can edit each and every somatic (body) cell in the body, you can cure the person. That's a tad bit difficult, to be honest. However, if the patient were a foetus, then we wouldn't need to make those many edits. However, if the gonadal (sex) cells of the body are edited, the future offspring of the patient would not have the condition. You're going to have to be a bit more specific with this 'condition'. | |
| May 29, 2016 at 5:22 | review | Suggested edits | |||
| S May 29, 2016 at 14:42 | |||||
| May 29, 2016 at 3:51 | review | First posts | |||
| May 29, 2016 at 5:23 | |||||
| May 29, 2016 at 3:49 | history | asked | user24161 | CC BY-SA 3.0 |