Gene Therapy Explained
ฝัง
- เผยแพร่เมื่อ 27 พ.ย. 2016
- Many diseases have a genetic basis, which means that the disease is caused by mutated genes which provide incorrect instructions that cause the cell to produce abnormal protein, or disable production of the protein completely.
In gene therapy, a functional copy of the gene is delivered into a patient’s own cells. Normal protein that is produced from a functional gene corrects the underlying cause of the disease, and may provide restorative disease-modifying effects.
Gene therapy uses engineered viruses to deliver genes into cells, such as adeno-associated virus (AAV) vectors, which are especially well suited for treating retinal diseases. Genetic defects in the eye can cause vision loss or blindness, and gene therapy can provide a long-term therapeutic benefit.
This video explains how gene therapy, delivered by AAV vectors, works.
For more info, visit www.agtc.com. - วิทยาศาสตร์และเทคโนโลยี
Writing a paper for BIO. Had to watch like 10 times.
THIS UGH :/ is it just me or is this video bad at explaining? the monotone and voice makes it SO hard to pay attention, and the visuals, while interesting, feel…cold and kind of sterile? It feels like a commercial (though, I guess it is lol!)
see you in medical school!
I am studying 11th-grade biology, from childhood I was interested in genetic engineering, I wanted to become GE, and I am researching gene therapy.... And after watching this video i would like to tell actg channel to upload more videos.. Thanks
I also like biology & want to be a GE . Ty sister
i am in 9th grade and i get this topic in detail haha
Wow this is so good to hear, hope you started majoring in genetic engineering
@@josyv4579 thank you so much☺i'm doing microbiology as a major stream and in a 2nd year...hope so I'll do genetics in master's degree
@@poppyshaikh1300 I wish you all the best ❤❤
I have a form of this called Rod Monochromatism so I really hope that one day this could treat my disease. I'm so happy that there are people actually working on a cure for me and others like me
try ageloc
Gene therapy is becoming a major field of research recently. We discuss these types of things over on this biotechnology server here. Feel free to join in. discord.gg/ftSbffu
old up there buddy imma help ppl like ya one day
Say true
Tricky....you'd have to design a vector to discriminate between cone types to deliver the correct genes to the right cell
Great video, explains gene therapy so well for us mere mortals.
You say “mere mortal” like you’re some whiny emo.
I'm a mere Mortal :p
#droigiangbe you are the best herbalist online no doubts. After using your herbal medication and getting cured I believed you for life and immediately my trust grew up more for you. I sincerely believe lots of people gonna despise you and blaspheme you for no reasons but they don’t know what they’re missing. You’re a powerful healer of herpes. You be my doctor forevermore for life. I rest my case . contact #droigiangbe on TH-cam'''''
Great video.. what softwares or apps do you use to make such animations? Its so good and detailed..
Explained really well
It just so happens I’m writing a research paper on gene therapy for my microbiology class... extremely glad I stumbled across this lol
Hello
The music in this video is so pleasant
Very interesting, i was very sceptic about sub retinal injections for gene therapy, i thought it is very traumatic. But after i seen achromatic dog "before" and "äfter"... im very impressed.. thank you
Now this, this is EPIC!
Wonderful video 😊 loved it
How does it work with alleles? If population X and population Y has different alleles, will gene therapy like this affect them both or is it allele-specific?
Thank you !
Is it works on incurable eye disease like stargardt disease ?How many times take this treatment for cure to stargardt or muscular dystrophy.?
What type of genes as far u know has a treatment or improvement..
Really looking forward to your ACHM studies. I am myself an achromatic that is currently participating in a study to treat the CNGB# ache version.
Alfred Lindeborg How is it going?
What education did you have to do to become that?
Alfred How is it going??is their any progress?
Cobid 19 Vaxine.
(Misspelled for obvious reasons)
Thx ...for concept clear...👍🏻👍🏻
Can AAV be a possible prognosis for melanoma therapy please???
Is possible gene therapy for eye optic nerve damage treatment for regeneration..
What should i study to have a career in gene therapy? Currently i am a bachelor in biotechnology at a small university
Covid-19 brought me here
Is the vaccines gene therapy? I was doing research and they seem very similar
Yes that vaccine is definitely gene therapy
Me too smh. The vaccine is gene therapy indeed
Maybe I should get another one, I think I have childhood dementia so need that gene therapy 👍👍
Can we not inject proteins instead? Or what if the retina disease isn't due to flawed genes?
How to manufacture the functional gene in labs?
In India available any branch agtc
which hospital can provide information with gene theraphy my son has terrible x-ald
i am searching solution
Cool I love it
How effective would this be to sarcopenia
Nice creative video
I have a genetic mutation on gene ADPRHL2 . Please specify the complementary DNA of mutation :
ADPRHL2 gene : (NM_017825.2) c.[14C>T];[14C>T] (p.[Ala5Val];[Ala5Val])
Many thanks. It helps for CRISPR .
Please find a gene therapy for keratoconus 😭 it hurts my eyes so bad 😔
Thankyou
Could a blood transfusion that produces the correct protein cure a mutated gene. And visa versa ?
Wonderful.....,
$AGTC will be a game changer. Got some shares at $3.95 in Jan before the surge. Lots of 'eyeballs' are on this company now. AGTC FTW!
LESSGGOOO BUY THE DIP!
What’s success rate for CNGA 3 genes??
Muscular dystrofe treatment bhe ?
TH-cam have dh12 gene mutation so I can participate in clinical trial I am staying in India CEO at present there is any treatment for th 12 gene mutation
Think also of sicklers, people with sickle cell disease as well, its a red blood cell disease that is like veey common in oill rich region of saudi in eastern province, common and sickle cell like has no real cure so gene therapy of bone marrow is advised
the revolution of science
So im guessing dont question the science behind this at all? K got it
Yep, just take your jabs every 6 months for the rest of your life & dont question it too much!
covid 19 vax brought me here
Is the vaccines gene therapy? I was doing research and they seem very similar
I have 22q deletion syndrome which means I dont have the proteins that make cells, which means i have had a cleft palate, speech disorder, neurodevelopmental disorder. Im hoping to get gene therapy soon.
I have wilson disease
this is cool I like this stock
I suffer from LCA3 disease and my vision is poor. Can gene therapy help me?
I'm from Pakistan. I have Retinitis Pigmentosa. How can I get treatment?
Wow👏🏻👏🏻👏🏻
I'm muscular atrophy type c patient please help me
suffer from LCA3 disease and my vision is poor. Can gene therapy help me?

This new gene that is inserted in the cell does not actually integrate itself in the cell's genome right? Otherwise that would be CRISPR-Cas9. The gene sits besides the genome but still has functional benefits but that would mean that the faulty gene would still be able to be passed on to the next generation? Germline editing is still illegal from what I know.
33 yo Man with XLRS. Proud of you, guys...
Transgenic organisms
Nice expression
please do more New researches using CRISPER for all genes
CRIPSR and gene therapy are separate techniques. Gene therapy involves insertion of fully functional genes for incorporation, while CRISPR edits original nucleotide sequences.
CRISPR
could it work with neural diseases like SMA ?
Yes it has shown to work for SMA in mice and a company has already filed for FDA approval . Google "SMA gene therapy mice"
the virus is engineered before or after harvesting them, sir?
During... A cell such as hek 239 has the virus genes to make the protein shell...and synthetic DNA is introduced to become enveloped by the protein shell. This is then harvested as the final product.
R.I.P AGTC
I have a question
If genes made the body's proteins, then from where come other body structures such as sugar and fat?
Fat is not made by the human body, it is stored after ingesting and digesting food. Sugar is made of glucose which is a product of respiration.
Umbrella corps: "that down write that down"
AGTC is very good, best information, but i cant understand english perfect. (but that okay 👍)
Only after reproduction. Reproduction could possible effect dna in ways we will not be able to predict.
Custom Gene Therapy: engineered from the patient's own cells and CRISPERed to replace faulty code, takes a few million $ in equipment and a team of super experts.
Available for those who "know someone", and can donate a 10 million dollars to the "researchers".
For the common folk, "yeah, we're working on it"; means "its not cheap enough yet for YOU"
Are the gene modifications inheritable?
Jakob Kirbach Good Question!... wanted an answer to this myself
They wouldn’t be as the transduction is local to the eye. If it was local to the testes then it would be inheritable as the sperm cells would carry the modifications whereas in this case only the cone cells do. Hope this helps.
The background music is extremely unneeded and distracting from trying to listen to the narrator. I'm trying to learn this stuff for a class, and I really wish I could turn the music off.
I wonder how dey got to know all these
Years of study on top of decades and decades of other peoples lifetime research. New technological advancements in lab instruments and computers, etc etc.
I just did not understand how you could fix a mutation just by changing one cell. Would its dna take over all the other cells like it?
It's a virus, it duplicates in your cells and spreads to surrounding cells. It eventually infects all the cells and mutates them according to their coding.
Thanks
its finally happening
Heroes
their brand name is also the four nucleic bases of DNA
How rich do I have to be to get this tho??
Depends on where in the world you live
During gene therapy, we embed one duplicate of a typical allele for each cell that needs it. For what reasons don't we embed an entire chromosome from a sound patient?
#droigiangbe you are the best herbalist online no doubts. After using your herbal medication and getting cured I believed you for life and immediately my trust grew up more for you. I sincerely believe lots of people gonna despise you and blaspheme you for no reasons but they don’t know what they’re missing. You’re a powerful healer of herpes. You be my doctor forevermore for life. I rest my case . contact #droigiangbe on TH-cam'''''
I'm grateful to have come across you Dr Igudia youtube, I'm now free after I used the medicine you sent me, having Herpes almost ruined my life but I'm happy today after I used your treatment.
Intellectual property portfolio? In other words you want to monopolize medicine?
success rate in clinical trials ?
HS Ayoub close to 100%. The only problem is, that sometimes mistakes can happen and there isn’t enough research on the safety yet. You meight get cancer from current gene therapy
@@CaptivaLP Use Artificial Intelligent near perfect Robots for operation.
Has this been approved by the FDA?
Yes it has!
@@CrazziiDukuu174 can this cure Myopia?
Samuel Christian No. Myopia is basically short sightedness which is corrected by wearing glasses.
@@CrazziiDukuu174 but isn’t it also a genetic eror?
Did I heard that correctly? “Engineered viruses” …
💥
STS brought me here.
Hello BSED Fil2A
All types of sma…not only 1
How genes are inserted into cancer cells ?
-kindly help me pls 😊
Using viral vectors
They inject a modified virus.
And it does it's job.
I am color blindness, strong protan since birth. I’ve never experinced full colors since birth and waited the gene therapy since monkeys trial in 2009. I couldn’t cook well, see the sunset, trafic light, match the dress, get dream jobs, and etc. I am fully disable honestly althogh it is not threatining condition. But most scientists dont understand how broken I am. They would rather find the cure of AMD and RP than CVD. In Indonesia said “sampai kapan ini berakhir ya Yesus?” Or in english “till when it will end oh JESUS?” I want to be cured like You ever did for blind people in Yerikho. Yeah, only hope can save my life now.
Everything happen for a reason. Don't feel bad. Even if you can't differ color one day everything will be changed. Be happy.
Why would anyone want to get this into their bodies for a viru s that you might get no serious symptoms of. This is more serious than they are showing it to be.
well nobody force you to take gene therapy, some people wanna see better
@@ravenn8600 looks at pfizer mrna "vaccine"
Dedicated to MicroBiologists,
Bio Technologists.
What happen in now
$AGTC, the best stock to BUY right now!
0:09-2:10 Something for me ignore this
The virus part of this freaks me out
What's wrong with injecting herpes in your eyes?
Jesus Christ well said Jesus
Harmless virus. Same as one plant is toxic to eat but another is edible. Both plants, but very different effects on human body.
The risky thing is unless the gene coding is perfected, we could one day cause a doomsday mutation just because a single gene is out of place. *Made a zombie strain. Oopsie!*
What about gene therapy after 7 years, or is it just theories and computer graphics?
The music is quite annoying but good video otherwise
*if you want a job look for team stellar on facebook, we are starting up, but if you want to study gene therapy with us, i need a big, big science team - we share findings*
Hi my son 7 years old he is dmd
Same condition my son 7year old he is dmd😔
Svend
i m suffring lgmd
2:16👆🏽dementia patients pay me now
I want to use this technology to create the first genetic sex change. I could make millions!
All Encompassing Evil da fuq?
Terrible audio mix.