uniQure

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@ericamadobegines8764 2 ай бұрын

The most beautiful video I have ever seen on this topic. Such a piece of art!!! 💌💌💌💌💌

@JannatiKhatun-e5b 3 ай бұрын

Johnson Kimberly Wilson Jessica Davis Jason

@pacific_marten 4 ай бұрын

Says capsid is shed inside the nucleus but video shows the capsid docked at a nuclear pore complex and "injecting" its genome into the nucleoplasm. It's also a double stranded molecule but the dsDNA synthesis occurs during / after capsid disassembly using the single stranded DNA viral genome (though, it's possible to package AAV with dsDNA).

@EaterGreen 7 ай бұрын

Could you make this contagious? like a disease that forces peoples genes to change?

@honsool3269 8 ай бұрын

Amazing how life is made

@hjpev6469 11 ай бұрын

This video acts like non-integration into the host genome is a benefit. But if you're trying to correct a faulty gene, why WOULDN'T you want the theraputic gene integrated into the host genome? Without that you're only giving someone a temporary treatment. Good for revenue maybe, but bad for the patient.

@mol_biologist30 11 ай бұрын

I don't understand. Please elaborate on that. Why do you think integrating the foreign DNA into the host genome would be beneficial?

@CamCorters 11 ай бұрын

Because otherwise how is this a long term solution to a disease? How can you ensure "stable and long term expression" of the targeted protein if the episomes aren't passed on when the cell divides? Would it no only stay in cells non-dividing cells? Either this would require routine follow up therapies or there is some mechanism not explained in the video that causes long term expression of the proteins? Could still be useful but the video states "stable and long term expression" of the molecule@@mol_biologist30

@littlemochi4440 11 ай бұрын

it's because AAV are small vector with a packaging capacity of 5kb and the problem is the immune system gradually neutralize AAV with antibodies. however, the good thing is that even though they are not permanent, AAV have a long-term expression of the target gene.

@Rose1999.x 11 ай бұрын

in some cases we are inducing for example pancreatic acinar cells into becoming beta cells, and in that case we wouldn't want every single pancreatic acinar cell to transcribe those genes and become beta cells now wouldn't we?

@lujasoninc. 9 ай бұрын

In the case of permanent or stable correction, another vector *LENTIVIRUS* or retroviruses can be used as they are capable of integrating into the host genome. However, human genome integration (patterns, sites and its effects) is still not yet fully understood... Therefore it is safe to first go easy "temporary" until some crucial questions are answered satisfactorily.

@sharongarcia4157 Жыл бұрын

Should I trust medical product like this cause I've went without all of it and I seem to be fine but idk

@cvspvr 10 ай бұрын

obviously, you'd only need this if you're sick

@prdevisleorleandre1337 10 ай бұрын

If you don't have genetic pathology, why do you want to use this kind of treatment ?

@itsmaanel Жыл бұрын

This is a work of art 😍❤

@amyjames3096 Жыл бұрын

Please post video updates

@FSK1138 Жыл бұрын

can gene therapy be used to increse Neurogenesis ?

@FSK1138 Жыл бұрын

the next 100 years is going to be amazing .. don't buy pills ,, just edit your genes

@Wheres-my-toes-bro Жыл бұрын

1:35 instead of a therapeutic molecule imagine it produces a cytotoxic synthetic protein that triggers inflammatory responses and autoimmune issues and no long term data on effects.

@life_2022 2 жыл бұрын

it forms episome like a second DNA not integrated cells own DNA if there is a missing gene it may work but if cells own DNA produce faulty protein or wrong protein it is going to build up in body episome will create right protein and at he same time cell's own dna will create wrong protein both

@shayanghafary6857 2 жыл бұрын

the animations here are insane! Thank you, very useful to see the whole process animated like this, very educational. helps understand.

@taharshahin8834 2 жыл бұрын

What are the latest developments in the drug statement for Huntington's patients?

@taharshahin8834 2 жыл бұрын

What are the latest developments in the drug statement for Huntington's patients?

@jessicarainey462 2 жыл бұрын

can you cite the research you're summarizing?

@shawonmondol2601 2 жыл бұрын

I am waiting for a great news ।

@cheeyeanwong600 2 жыл бұрын

Are there any updates on AMT-150 initiation of IND?

@jaredbrito755 3 жыл бұрын

You guys are about to save my life and the life of so many others. Keep up the good work!

@jacklynallen7549 3 жыл бұрын

Does anyone know what company produced this animation?!

@samanthadowning1414 3 жыл бұрын

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@viderethevaccinatorfromhol7536 3 жыл бұрын

This is probably the only method for many of my fellow patient's to save our lives. So hurry up a bit please 😉 after so many disappointments this year we can definitely use some good news.

@timmie9975 3 жыл бұрын

We are trying our best! <3

@learningpower9437 3 жыл бұрын

Excellent video .... !!!! If m-RNA is formed from the transcription of the DNA episome ... what will happen when the cells that have the episomes die ...? .... Do we have to re-inject the patient ...? ... But you will need a different adenovirus ... because for the first one ... the body will have a strong immune response to it ... Maybe a retrovirus ... will solve the problem ...

@underzepump2905 2 жыл бұрын

Regarding your first question: this depends on the target cells. Trials are in place in a number of therapy areas looking at duration of response. Presumably, if the response wanes after a period, another injection or infusion would be required... Which brings us to the next question - will the same AAV work again, even when the patient has neutralising antibodies? Some trials are looking at this, and it looks like the answer is "it depends" at the moment (on which AAV serotype is used, the individual patient and their pre-existing neutralising antibodies, and what level of response is expected/desired).

@nzehavi 3 жыл бұрын

what an amazing animation!!!!

@XxSolvexX 4 жыл бұрын

Exceptional animations. Amazing how seeing it like this really settles in the feeling of just how incredible this technology is to manipulate our already unbelievable physiological mechanisms

@cl9315 4 жыл бұрын

When will this treatment be available for people who suffer from this horrific disease?

@viderethevaccinatorfromhol7536 3 жыл бұрын

It will take years unfortunately. These fase 1/2 trials take at least 5years. A treatment will take a few years more. I am sorry but it's a fact. I have HD and following all scientific developments across the planet. For my generation it's a bit to late, but I am very positive for the next generation.

@shuatastic 3 жыл бұрын

@@viderethevaccinatorfromhol7536 I am sorry that it is unlikely these therapies will be unavailable for you. Some good news is WAVE is restarting its trial and PROOF-HD is in phase 3. So there is potential good news for you depending on your stage of the disease! I am also following everything all over the planet as my wife has the gene (we found out because she knows it runs in the family and we are trying to family plan). Thankfully she is not symptomatic and won't be for at least 15 years, which gives us some time for this horrible disease to receive some breakthroughs. I think my wife is a little tired of me looking at everything under the sun when she isn't even symptomatic but it weirdly helps me relax that there's so much hope in the pipeline.

@emilylauriana4619 2 жыл бұрын

@@shuatastic i am happy to read this comment, you are very generous in trying to help your wife, you really love her. l try to be positive my family on father's side has this terrible DH, my father has the symptoms he is 58 years old, have faith that we will have a cure soon. We have many treatments being developed, I believe there will be a cure soon.

@daoyuzhang1648 4 жыл бұрын

harvardtothebighouse.com/2020/01/31/logistical-and-technical-analysis-of-the-origins-of-the-wuhan-coronavirus-2019-ncov/ Intentional or Incidental Serial passage of animal-derived pathogens in human cells is dangerous-human cell cultures have no immune systems, immune systems that would have eliminated any animal-sourced viruses before they could even distantly adapt to the human physiology. Without a immune system the pathogens could easily adapt to infecting human cells during culture passage. Often to ridiculous levels as seen in SARS-CoV-2 as the much simpler selection landscape selects for the highest possible binding affinity of the virus surface proteins to the specific receptor(s) expressed on the cells, without other concerns like host-virus interaction, population and behavior of the host animal or immune responses that would have limited the binding affinity in the wild. Potential pandemic zoonotic pathogens could be easily generated from such passages, that could happen even during the isolation process of the viruses themselves. Better cloning for a cure rather than a new potentially deadly pathogen, like what the USAMRRID/ Pentagon have done for the past three decades www.researchgate.net/publication/340786428_COVID-19_Pandemic_Its_Origin_Implications_and_Treatments Cloning the antibodies of recovered patients could be used to easily generate a large amount of life-saving, side-effects free cure for the ill, and the gene and plasmids themselves could be used as a vaccine.