Gene Therapy Explained
Ғылым және технология
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.
Пікірлер: 191
Writing a paper for BIO. Had to watch like 10 times.
@user-vd8nm8sv6x
3 жыл бұрын
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!)
@klam77
2 жыл бұрын
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
@BikashDas-hm6zg
4 жыл бұрын
I also like biology & want to be a GE . Ty sister
@ms-finearts5186
3 жыл бұрын
i am in 9th grade and i get this topic in detail haha
@josyv4579
2 жыл бұрын
Wow this is so good to hear, hope you started majoring in genetic engineering
@poppyshaikh1300
2 жыл бұрын
@@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
@josyv4579
2 жыл бұрын
@@poppyshaikh1300 I wish you all the best ❤❤
Great video, explains gene therapy so well for us mere mortals.
@Luigi_Mario_1997
3 жыл бұрын
You say “mere mortal” like you’re some whiny emo.
@navinsawesomeanddivineacco4221
3 жыл бұрын
I'm a mere Mortal :p
@usainiyahuza8034
Жыл бұрын
#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 KZread'''''
It just so happens I’m writing a research paper on gene therapy for my microbiology class... extremely glad I stumbled across this lol
@sumaiyasulaiman35
5 ай бұрын
Hello
Great video.. what softwares or apps do you use to make such animations? Its so good and detailed..
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
@joeyvillanueva4607
7 жыл бұрын
try ageloc
@warioland523
6 жыл бұрын
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
@pmz558
6 жыл бұрын
old up there buddy imma help ppl like ya one day
@biologyhub94439
5 жыл бұрын
Say true
@brainwashedbyevidence948
4 жыл бұрын
Tricky....you'd have to design a vector to discriminate between cone types to deliver the correct genes to the right cell
The music in this video is so pleasant
Explained really well
What type of genes as far u know has a treatment or improvement..
Is it works on incurable eye disease like stargardt disease ?How many times take this treatment for cure to stargardt or muscular dystrophy.?
Now this, this is EPIC!
What should i study to have a career in gene therapy? Currently i am a bachelor in biotechnology at a small university
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?
Can AAV be a possible prognosis for melanoma therapy please???
Wonderful video 😊 loved it
Thank you !
Cobid 19 Vaxine. (Misspelled for obvious reasons)
How to manufacture the functional gene in labs?
Is possible gene therapy for eye optic nerve damage treatment for regeneration..
How effective would this be to sarcopenia
Thx ...for concept clear...👍🏻👍🏻
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
What’s success rate for CNGA 3 genes??
Can we not inject proteins instead? Or what if the retina disease isn't due to flawed genes?
Muscular dystrofe treatment bhe ?
Could a blood transfusion that produces the correct protein cure a mutated gene. And visa versa ?
which hospital can provide information with gene theraphy my son has terrible x-ald i am searching solution
KZread 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
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 .
In India available any branch agtc
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.
@Kurt_707
7 жыл бұрын
Alfred Lindeborg How is it going?
@ilinca9194
6 жыл бұрын
What education did you have to do to become that?
@beenishkhalid1224
5 жыл бұрын
Alfred How is it going??is their any progress?
Nice creative video
Please find a gene therapy for keratoconus 😭 it hurts my eyes so bad 😔
I suffer from LCA3 disease and my vision is poor. Can gene therapy help me?
33 yo Man with XLRS. Proud of you, guys...
@Rswinters1
7 жыл бұрын
Transgenic organisms
the revolution of science
I'm from Pakistan. I have Retinitis Pigmentosa. How can I get treatment?
suffer from LCA3 disease and my vision is poor. Can gene therapy help me? 
Thankyou
Covid-19 brought me here
@dablackxican5978
3 жыл бұрын
Is the vaccines gene therapy? I was doing research and they seem very similar
@whyseman375
3 жыл бұрын
Yes that vaccine is definitely gene therapy
@AccordingToArmon
2 жыл бұрын
Me too smh. The vaccine is gene therapy indeed
@TheHeroWhoAsked
Жыл бұрын
Maybe I should get another one, I think I have childhood dementia so need that gene therapy 👍👍
$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!
@kawhi-theking_of_the_north3536
3 жыл бұрын
LESSGGOOO BUY THE DIP!
I'm muscular atrophy type c patient please help me
could it work with neural diseases like SMA ?
@Kevin-qy9wf
5 жыл бұрын
Yes it has shown to work for SMA in mice and a company has already filed for FDA approval . Google "SMA gene therapy mice"
R.I.P AGTC
Wonderful.....,
Cool I love it
the virus is engineered before or after harvesting them, sir?
@brainwashedbyevidence948
4 жыл бұрын
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.
Umbrella corps: "that down write that down"
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.
@Shobha-xw7sk
6 ай бұрын
I have wilson disease
this is cool I like this stock
I have a question If genes made the body's proteins, then from where come other body structures such as sugar and fat?
@aliyachoudhury8420
3 жыл бұрын
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.
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.
So im guessing dont question the science behind this at all? K got it
@XOXOX4242
2 жыл бұрын
Yep, just take your jabs every 6 months for the rest of your life & dont question it too much!
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
Nice expression
I wonder how dey got to know all these
@miathapapaya
6 жыл бұрын
Years of study on top of decades and decades of other peoples lifetime research. New technological advancements in lab instruments and computers, etc etc.
Wow👏🏻👏🏻👏🏻
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.
please do more New researches using CRISPER for all genes
@DTGMRuns
5 жыл бұрын
CRIPSR and gene therapy are separate techniques. Gene therapy involves insertion of fully functional genes for incorporation, while CRISPR edits original nucleotide sequences.
@sagudanicasnicas2660
5 жыл бұрын
CRISPR
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"
its finally happening
Are the gene modifications inheritable?
@louisbroadway4392
4 жыл бұрын
Jakob Kirbach Good Question!... wanted an answer to this myself
@willgreen8310
4 жыл бұрын
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.
How rich do I have to be to get this tho??
@CrazziiDukuu174
3 жыл бұрын
Depends on where in the world you live
Only after reproduction. Reproduction could possible effect dna in ways we will not be able to predict.
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?
@bodypillow3800
5 жыл бұрын
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.
@life-kk9xu
5 жыл бұрын
Thanks
AGTC is very good, best information, but i cant understand english perfect. (but that okay 👍)
their brand name is also the four nucleic bases of DNA
success rate in clinical trials ?
@CaptivaLP
5 жыл бұрын
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
@fireartdrawer9531
4 жыл бұрын
@@CaptivaLP Use Artificial Intelligent near perfect Robots for operation.
Intellectual property portfolio? In other words you want to monopolize medicine?
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?
@usainiyahuza8034
Жыл бұрын
#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 KZread'''''
Did I heard that correctly? “Engineered viruses” …
Heroes
Has this been approved by the FDA?
@CrazziiDukuu174
3 жыл бұрын
Yes it has!
@samuelzev4076
3 жыл бұрын
@@CrazziiDukuu174 can this cure Myopia?
@CrazziiDukuu174
3 жыл бұрын
Samuel Christian No. Myopia is basically short sightedness which is corrected by wearing glasses.
@samuelzev4076
3 жыл бұрын
@@CrazziiDukuu174 but isn’t it also a genetic eror?
All types of sma…not only 1
Dedicated to MicroBiologists, Bio Technologists.
💥
covid 19 vax brought me here
@dablackxican5978
3 жыл бұрын
Is the vaccines gene therapy? I was doing research and they seem very similar
How genes are inserted into cancer cells ? -kindly help me pls 😊
@twowhitecircleswithgraybac3376
4 жыл бұрын
Using viral vectors
@ras573
3 жыл бұрын
They inject a modified virus. And it does it's job.
What happen in now
0:09-2:10 Something for me ignore this
STS brought me here. Hello BSED Fil2A
$AGTC, the best stock to BUY right now!
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.
The virus part of this freaks me out
@mrdanker6756
6 жыл бұрын
What's wrong with injecting herpes in your eyes?
@pmz558
6 жыл бұрын
Jesus Christ well said Jesus
@miathapapaya
6 жыл бұрын
Harmless virus. Same as one plant is toxic to eat but another is edible. Both plants, but very different effects on human body.
@LightningSe7en
5 жыл бұрын
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!*
The music is quite annoying but good video otherwise
What about gene therapy after 7 years, or is it just theories and computer graphics?
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.
@solyanafekadu
2 ай бұрын
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.
@ravenn8600
2 жыл бұрын
well nobody force you to take gene therapy, some people wanna see better
@hunterbiden4975
Жыл бұрын
@@ravenn8600 looks at pfizer mrna "vaccine"
Hi my son 7 years old he is dmd
@Aararadhykokate88
3 жыл бұрын
Same condition my son 7year old he is dmd😔
*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*
Svend
i m suffring lgmd
2:16👆🏽dementia patients pay me now
Terrible audio mix.
I want to use this technology to create the first genetic sex change. I could make millions!
@gopherlee9427
6 жыл бұрын
All Encompassing Evil da fuq?