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Message
re: Human Gene Therapy
Posted on 5/20/14 at 10:28 pm to Hater Bait
Posted on 5/20/14 at 10:28 pm to Hater Bait
quote:
So, you're saying a virus can help my body "kick start" and produce this gene?
Missing genes can be installed into a given genome. It simply requires a safe region of your DNA with active promoter and enhancer sequences. If the gene is installed in such a region, it will be actively transcribed.
There are a multitude of techniques currently being studied with respect to genome editing, many of which don't involve viruses. We have the ability to remove genes, modify defective genes, and even delete genes. All of this has been accomplished to some degree in animal models.
This post was edited on 5/20/14 at 10:30 pm
1
Posted on 5/20/14 at 10:31 pm to Cs
The other part that makes it harder is the timeline of the gene insertion. i.e. will simply replacing a protein correct/improve the problem or has someone already missed out on the appropriate developmental steps as a result of it since so many things on the cellular level are orchestrated by the local milieu (not speaking to your case in specific as I'm really not familiar with retinoschisis, I'm not an optho).
Really excited to see where gene replacement therapy takes us in the future but anticipate it will be like most other things where we have plenty of unintended consequences that we learn even more from. It's how science works.
Really excited to see where gene replacement therapy takes us in the future but anticipate it will be like most other things where we have plenty of unintended consequences that we learn even more from. It's how science works.
This post was edited on 5/20/14 at 10:35 pm
Posted on 5/22/14 at 1:27 am to TMDawg
Thank you to all.
Your answers gives me a lot of hope.
Your answers gives me a lot of hope.
Posted on 5/22/14 at 3:42 am to Hater Bait
FWIW, this subject is what the latest Bourne Identity movie was about. Lots of knowledge dropped if you watch for it.
By the way, Matt Damon was not in the movie. Not even for a second. Weird.
By the way, Matt Damon was not in the movie. Not even for a second. Weird.
Posted on 5/22/14 at 7:56 am to AtlantaLSUfan
The other sad part about all this is that large drug companies and research firms are really only focused on a few cases. They dont spend anytime researchiing specific cases like yours, or my son's, who has Cystic Fibrosis. Thankfully the CF Foundation does their own and have made huge strides in the last 10 years. They've isolated the specific protein that causes CF but there are over 1500 different mutations of that protein (CFTR). With only 70,000 people worldwide having CF, its not a very profitable business for the pharm companies.
The future of gene replacement is only going to grow and get better. The things they can do now are amazing. It takes money, and lots of it from idividuals like you and I though. Hang in there!
The future of gene replacement is only going to grow and get better. The things they can do now are amazing. It takes money, and lots of it from idividuals like you and I though. Hang in there!
Posted on 5/22/14 at 8:18 am to Hater Bait
Pretty much how the zombie apocalypse is going to start
ETA: Hater Bait = Patient 0
ETA: Hater Bait = Patient 0
This post was edited on 5/22/14 at 8:19 am
Posted on 5/22/14 at 8:47 am to RockCityHog
quote:
my son's, who has Cystic Fibrosis
Very sorry to hear about your son's disease, thoughts with you and your family on that.
I know the pain of CF, my brother died after battling CF for 24 years. He was my best friend.
Posted on 5/22/14 at 10:26 am to LobbyingLeprechaun
We try to be very optimistic about it but knowing its terminal still doesnt help. They made lots of progress and the life expectancy is around 37 now but thats still not good enough for my son! We will continue to fight for him and give him every opportunity we can to live a normal life. We hope he'll be a champion for his own cause as well. Very sorry to hear about your brother. We've met and heard lts of stories from other CF families. It just plain sucks. ANd just because his isnt bad right now doesn't mean it wont get worse.
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