Gene Therapy Explained

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  • Опубликовано: 27 ноя 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.
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Комментарии • 191

  • @el_engineer0p106
    @el_engineer0p106 3 года назад +51

    Writing a paper for BIO. Had to watch like 10 times.

    • @user-vd8nm8sv6x
      @user-vd8nm8sv6x 3 года назад +2

      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
      @klam77 2 года назад

      see you in medical school!

  • @poppyshaikh1300
    @poppyshaikh1300 5 лет назад +66

    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
      @BikashDas-hm6zg 4 года назад +1

      I also like biology & want to be a GE . Ty sister

    • @ms-finearts5186
      @ms-finearts5186 3 года назад +1

      i am in 9th grade and i get this topic in detail haha

    • @josyv4579
      @josyv4579 2 года назад +1

      Wow this is so good to hear, hope you started majoring in genetic engineering

    • @poppyshaikh1300
      @poppyshaikh1300 2 года назад +4

      @@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
      @josyv4579 2 года назад +1

      @@poppyshaikh1300 I wish you all the best ❤❤

  • @Kurt_707
    @Kurt_707 7 лет назад +79

    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
      @joeyvillanueva4607 7 лет назад +1

      try ageloc

    • @warioland523
      @warioland523 6 лет назад

      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
      @pmz558 6 лет назад

      old up there buddy imma help ppl like ya one day

    • @biologyhub94439
      @biologyhub94439 5 лет назад

      Say true

    • @brainwashedbyevidence948
      @brainwashedbyevidence948 4 года назад

      Tricky....you'd have to design a vector to discriminate between cone types to deliver the correct genes to the right cell

  • @ginosanchez7
    @ginosanchez7 5 лет назад +8

    It just so happens I’m writing a research paper on gene therapy for my microbiology class... extremely glad I stumbled across this lol

  • @TAMOGHNADASBBT
    @TAMOGHNADASBBT 5 лет назад +6

    Great video.. what softwares or apps do you use to make such animations? Its so good and detailed..

  • @shelleysmith9709
    @shelleysmith9709 5 лет назад +63

    Great video, explains gene therapy so well for us mere mortals.

    • @Luigi_Mario_1997
      @Luigi_Mario_1997 3 года назад +1

      You say “mere mortal” like you’re some whiny emo.

    • @navinsawesomeanddivineacco4221
      @navinsawesomeanddivineacco4221 3 года назад

      I'm a mere Mortal :p

    • @usainiyahuza8034
      @usainiyahuza8034 Год назад

      #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 RUclips'''''

  • @Zzz21140
    @Zzz21140 7 месяцев назад +2

    Explained really well

  • @zahraiqba765
    @zahraiqba765 7 лет назад +35

    The music in this video is so pleasant

  • @joshireson5457
    @joshireson5457 5 лет назад +6

    Now this, this is EPIC!

  • @swapnadeepdey9394
    @swapnadeepdey9394 3 года назад +1

    Wonderful video 😊 loved it

  • @KortexYTT
    @KortexYTT 4 года назад +1

    Thank you !

  • @sharadpatil1837
    @sharadpatil1837 6 лет назад

    Is it works on incurable eye disease like stargardt disease ?How many times take this treatment for cure to stargardt or muscular dystrophy.?

  • @John-un3lj
    @John-un3lj 4 года назад

    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?

  • @osamasaid2210
    @osamasaid2210 6 лет назад

    What type of genes as far u know has a treatment or improvement..

  • @divyanshu2303
    @divyanshu2303 4 года назад

    Thx ...for concept clear...👍🏻👍🏻

  • @kr-1543
    @kr-1543 2 года назад +4

    Cobid 19 Vaxine.
    (Misspelled for obvious reasons)

  • @djanibianco3301
    @djanibianco3301 3 года назад +2

    What should i study to have a career in gene therapy? Currently i am a bachelor in biotechnology at a small university

  • @user-iy8pg4zc6b
    @user-iy8pg4zc6b 2 года назад +1

    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

  • @alfredlindeborg4736
    @alfredlindeborg4736 7 лет назад +5

    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
      @Kurt_707 7 лет назад

      Alfred Lindeborg How is it going?

    • @ilinca9194
      @ilinca9194 6 лет назад

      What education did you have to do to become that?

    • @beenishkhalid1224
      @beenishkhalid1224 5 лет назад

      Alfred How is it going??is their any progress?

  • @shanavasa3078
    @shanavasa3078 3 года назад

    Is possible gene therapy for eye optic nerve damage treatment for regeneration..

  • @WaleShozy
    @WaleShozy 2 года назад

    Can AAV be a possible prognosis for melanoma therapy please???

  • @yeshwanthreddy268
    @yeshwanthreddy268 2 года назад +1

    RUclips 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

  • @UHFStation1
    @UHFStation1 4 года назад

    Can we not inject proteins instead? Or what if the retina disease isn't due to flawed genes?

  • @valhallanews5675
    @valhallanews5675 3 года назад +18

    Covid-19 brought me here

    • @dablackxican5978
      @dablackxican5978 3 года назад +1

      Is the vaccines gene therapy? I was doing research and they seem very similar

    • @whyseman375
      @whyseman375 3 года назад +2

      Yes that vaccine is definitely gene therapy

    • @AccordingToArmon
      @AccordingToArmon 2 года назад

      Me too smh. The vaccine is gene therapy indeed

    • @TheHeroWhoAsked
      @TheHeroWhoAsked Год назад

      Maybe I should get another one, I think I have childhood dementia so need that gene therapy 👍👍

  • @sarahtlm013
    @sarahtlm013 3 года назад

    How to manufacture the functional gene in labs?

  • @sherlinefenelon818
    @sherlinefenelon818 3 года назад

    which hospital can provide information with gene theraphy my son has terrible x-ald
    i am searching solution

  • @shelbyjenkins1181
    @shelbyjenkins1181 Год назад +2

    Cool I love it

  • @biologyhub94439
    @biologyhub94439 5 лет назад +1

    Nice creative video

  • @rohanroy6629
    @rohanroy6629 3 года назад +1

    the revolution of science

  • @beenishkhalid1224
    @beenishkhalid1224 5 лет назад

    What’s success rate for CNGA 3 genes??

  • @mohammadzaid1023
    @mohammadzaid1023 5 месяцев назад

    In India available any branch agtc

  • @ashekhinahpt1197
    @ashekhinahpt1197 3 года назад

    Could a blood transfusion that produces the correct protein cure a mutated gene. And visa versa ?

  • @jeffreyeuro6523
    @jeffreyeuro6523 2 года назад

    How effective would this be to sarcopenia

  • @marklee3570
    @marklee3570 3 года назад +1

    $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!

  • @MegaDixen
    @MegaDixen 3 года назад

    this is cool I like this stock

  • @vipulaggarwal328
    @vipulaggarwal328 3 года назад

    Thankyou

  • @azharhussain3849
    @azharhussain3849 3 года назад

    Wonderful.....,

  • @maedehdelshad8551
    @maedehdelshad8551 Год назад

    I suffer from LCA3 disease and my vision is poor. Can gene therapy help me?

  • @enricopetrucci
    @enricopetrucci 7 лет назад +2

    33 yo Man with XLRS. Proud of you, guys...

  • @RuralChemist
    @RuralChemist 4 месяца назад +1

    R.I.P AGTC

  • @melissaalvarado94
    @melissaalvarado94 3 года назад +2

    Please find a gene therapy for keratoconus 😭 it hurts my eyes so bad 😔

  • @dogemoon5871
    @dogemoon5871 3 года назад +12

    So im guessing dont question the science behind this at all? K got it

    • @XOXOX4242
      @XOXOX4242 2 года назад +3

      Yep, just take your jabs every 6 months for the rest of your life & dont question it too much!

  • @arin5047
    @arin5047 Год назад

    Umbrella corps: "that down write that down"

  • @maedehdelshad8551
    @maedehdelshad8551 Год назад

    suffer from LCA3 disease and my vision is poor. Can gene therapy help me?

  • @sibu_puhal7255
    @sibu_puhal7255 9 месяцев назад

    I'm muscular atrophy type c patient please help me

  • @funny11744
    @funny11744 5 лет назад

    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 .

  • @surendrachaurasia1047
    @surendrachaurasia1047 6 лет назад +1

    Muscular dystrofe treatment bhe ?

  • @waterbird2686
    @waterbird2686 5 лет назад

    could it work with neural diseases like SMA ?

    • @Kevin-qy9wf
      @Kevin-qy9wf 5 лет назад

      Yes it has shown to work for SMA in mice and a company has already filed for FDA approval . Google "SMA gene therapy mice"

  • @zahraiqba765
    @zahraiqba765 7 лет назад +1

    Wow👏🏻👏🏻👏🏻

  • @ionescho
    @ionescho Год назад

    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.

  • @melaries1823
    @melaries1823 3 года назад +1

    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.

  • @owaisshaikh2144
    @owaisshaikh2144 3 года назад

    I'm from Pakistan. I have Retinitis Pigmentosa. How can I get treatment?

  • @dutamulia
    @dutamulia 4 года назад

    the virus is engineered before or after harvesting them, sir?

    • @brainwashedbyevidence948
      @brainwashedbyevidence948 4 года назад

      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.

  • @abhisheksahoo6863
    @abhisheksahoo6863 4 года назад

    Nice expression

  • @runsthroughmyveins8
    @runsthroughmyveins8 3 года назад +8

    covid 19 vax brought me here

    • @dablackxican5978
      @dablackxican5978 3 года назад

      Is the vaccines gene therapy? I was doing research and they seem very similar

  • @user-zf1ti3yg3v
    @user-zf1ti3yg3v 7 лет назад +1

    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

  • @itsme477
    @itsme477 5 лет назад +10

    please do more New researches using CRISPER for all genes

    • @DTGMRuns
      @DTGMRuns 5 лет назад +3

      CRIPSR and gene therapy are separate techniques. Gene therapy involves insertion of fully functional genes for incorporation, while CRISPR edits original nucleotide sequences.

    • @sagudanicasnicas2660
      @sagudanicasnicas2660 5 лет назад +3

      CRISPR

  • @solosailor222
    @solosailor222 3 года назад +1

    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"

  • @imanolk5513
    @imanolk5513 2 года назад +2

    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
      @ravenn8600 2 года назад

      well nobody force you to take gene therapy, some people wanna see better

    • @hunterbiden4975
      @hunterbiden4975 Год назад +2

      @@ravenn8600 looks at pfizer mrna "vaccine"

  • @Emily-yt1xx
    @Emily-yt1xx 2 года назад

    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.

  • @mohamedbouacida2722
    @mohamedbouacida2722 4 года назад

    I have a question
    If genes made the body's proteins, then from where come other body structures such as sugar and fat?

    • @aliyachoudhury8420
      @aliyachoudhury8420 3 года назад +1

      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.

  • @jraser2885
    @jraser2885 4 года назад +2

    Only after reproduction. Reproduction could possible effect dna in ways we will not be able to predict.

  • @adinatalahatova8123
    @adinatalahatova8123 5 лет назад

    AGTC is very good, best information, but i cant understand english perfect. (but that okay 👍)

  • @rik-keymusic160
    @rik-keymusic160 2 года назад +1

    Did I heard that correctly? “Engineered viruses” …

  • @Hugo-lu6jg
    @Hugo-lu6jg 5 лет назад +1

    its finally happening

  • @jakobkirbach7318
    @jakobkirbach7318 4 года назад

    Are the gene modifications inheritable?

    • @louisbroadway4392
      @louisbroadway4392 4 года назад +1

      Jakob Kirbach Good Question!... wanted an answer to this myself

    • @willgreen8310
      @willgreen8310 4 года назад +1

      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.

  • @nursepurity2955
    @nursepurity2955 6 лет назад +1

    I wonder how dey got to know all these

    • @miathapapaya
      @miathapapaya 6 лет назад +4

      Years of study on top of decades and decades of other peoples lifetime research. New technological advancements in lab instruments and computers, etc etc.

  • @maeranellarint7060
    @maeranellarint7060 3 года назад +1

    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
      @usainiyahuza8034 Год назад

      #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 RUclips'''''

  • @life-kk9xu
    @life-kk9xu 5 лет назад

    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
      @bodypillow3800 5 лет назад +3

      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
      @life-kk9xu 5 лет назад

      Thanks

  • @oubaybraougui7831
    @oubaybraougui7831 2 года назад

    their brand name is also the four nucleic bases of DNA

  • @HSAyoub
    @HSAyoub 6 лет назад +7

    success rate in clinical trials ?

    • @CaptivaLP
      @CaptivaLP 5 лет назад

      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
      @fireartdrawer9531 4 года назад

      @@CaptivaLP Use Artificial Intelligent near perfect Robots for operation.

  • @immigrantworld4899
    @immigrantworld4899 3 года назад

    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
      @solyanafekadu 2 месяца назад

      Everything happen for a reason. Don't feel bad. Even if you can't differ color one day everything will be changed. Be happy.

  • @jeepz669
    @jeepz669 3 года назад

    How rich do I have to be to get this tho??

  • @aang7505
    @aang7505 4 года назад +1

    Heroes

  • @UHFStation1
    @UHFStation1 4 года назад

    Intellectual property portfolio? In other words you want to monopolize medicine?

  • @janisnielson3529
    @janisnielson3529 8 месяцев назад

    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.

  • @ronbacani3742
    @ronbacani3742 3 года назад

    STS brought me here.
    Hello BSED Fil2A

  • @jacobinvesting5657
    @jacobinvesting5657 Год назад +1

    $AGTC, the best stock to BUY right now!

  • @DouglasJediKnight
    @DouglasJediKnight Год назад

    All types of sma…not only 1

  • @typicalrockhound9887
    @typicalrockhound9887 2 года назад

    💥

  • @gomathib2310
    @gomathib2310 3 года назад

    Dedicated to MicroBiologists,
    Bio Technologists.

  • @karmand879
    @karmand879 Год назад

    What happen in now

  • @user-st9qe3dh5t
    @user-st9qe3dh5t 6 месяцев назад

    What about gene therapy after 7 years, or is it just theories and computer graphics?

  • @matthewc4895
    @matthewc4895 5 лет назад

    0:09-2:10 Something for me ignore this

  • @samuelzev4076
    @samuelzev4076 3 года назад

    Has this been approved by the FDA?

    • @CrazziiDukuu174
      @CrazziiDukuu174 3 года назад +1

      Yes it has!

    • @samuelzev4076
      @samuelzev4076 3 года назад

      @@CrazziiDukuu174 can this cure Myopia?

    • @CrazziiDukuu174
      @CrazziiDukuu174 3 года назад

      Samuel Christian No. Myopia is basically short sightedness which is corrected by wearing glasses.

    • @samuelzev4076
      @samuelzev4076 3 года назад

      @@CrazziiDukuu174 but isn’t it also a genetic eror?

  • @kenw1849
    @kenw1849 Год назад

    The music is quite annoying but good video otherwise

  • @willankers2426
    @willankers2426 6 лет назад +2

    The virus part of this freaks me out

    • @mrdanker6756
      @mrdanker6756 6 лет назад

      What's wrong with injecting herpes in your eyes?

    • @pmz558
      @pmz558 6 лет назад +2

      Jesus Christ well said Jesus

    • @miathapapaya
      @miathapapaya 6 лет назад +5

      Harmless virus. Same as one plant is toxic to eat but another is edible. Both plants, but very different effects on human body.

    • @LightningSe7en
      @LightningSe7en 5 лет назад +1

      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!*

  • @melanievidal2578
    @melanievidal2578 4 года назад

    How genes are inserted into cancer cells ?
    -kindly help me pls 😊

  • @HAZNAUT
    @HAZNAUT 3 года назад +1

    *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*

  • @uzmaatif3209
    @uzmaatif3209 3 года назад

    Hi my son 7 years old he is dmd

  • @mathiaspetersen536
    @mathiaspetersen536 5 лет назад

    Svend

  • @ramasharma6113
    @ramasharma6113 5 лет назад

    i m suffring lgmd

  • @Heresheis0818
    @Heresheis0818 Год назад

    2:16👆🏽dementia patients pay me now

  • @techroach6343
    @techroach6343 3 года назад

    Terrible audio mix.

  • @allencompassingevil
    @allencompassingevil 6 лет назад

    I want to use this technology to create the first genetic sex change. I could make millions!