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uniQure
เข้าร่วมเมื่อ 19 ต.ค. 2016
uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B - an historic achievement based on more than a decade of research and clinical development - represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com
Patient Advocate Ashley Clarke - Getting involved in Huntington's disease advocacy work
UK-based HD Patient Advocate Ashley Clarke discusses her decision to get involved in Huntington's disease patient advocacy work, and the critical role of patient advocacy in disease awareness and education.
มุมมอง: 357
วีดีโอ
Huntington's Disease Patient Advocate Ashley Clarke - An HD Caregiver's Story
มุมมอง 2.1Kปีที่แล้ว
Ashley Clarke, a Huntington's disease patient advocate based in Northern Ireland, shares her experience as a caregiver for a parent with Huntington's disease, and discusses the urgent need for treatments for this devastating genetic disease.
Celebrating the International Day of Women & Girls in Science!
มุมมอง 58ปีที่แล้ว
Celebrating the International Day of Women & Girls in Science!
HD Community Advocates Charles & Jessi King
มุมมอง 248ปีที่แล้ว
As an inherited disease, Huntington’s disease (HD) can have a profound effect on families. When Charles when diagnosed with HD, he and his wife Jessi made the decision to become active in the community and to do whatever they can to help raise awareness and move research forward. “Every little bit helps,” says Charles. “Keep holding on to hope and hopefully we can get some treatments soon.”
uniQure's Passion for the Patient
มุมมอง 6632 ปีที่แล้ว
Senior Director of Global Patient Advocacy Dan Leonard discusses uniQure's patient focus and the work our Patient Advocacy group does with the patient communities we serve.
uniQure's Mission, Vision, and Values
มุมมอง 1.9K2 ปีที่แล้ว
CEO Matt Kapusta talks about uniQure's mission to transform the lives of patients through the power of genetics, and how that mission drives who we are at uniQure.
uniQure's miQURE Technology
มุมมอง 2.7K3 ปีที่แล้ว
This 3D animation showcases uniQure's miQURE gene-silencing technology.
AMT-130 Administration
มุมมอง 9K4 ปีที่แล้ว
A narrated animated illustration of how AMT-130 is administered into the caudate and striatum regions of the brain via a micro-catheter. Learn more about AMT-130, an experimental gene therapy for the treatment of Huntington's disease, here: uniqure.com/gene-therapy/huntingtons-disease.php.
Life at uniQure
มุมมอง 7424 ปีที่แล้ว
This 60-second b-roll clip features uniQure employees and locations and is available for media use.
uniQure 30-Second NASDAQ Marquee Video
มุมมอง 2594 ปีที่แล้ว
We were proud to have this video featuring uniQure employees and sites featured on the Nasdaq marquee in Times Square as we rang the Opening Bell on Tuesday, October 29, 2019.
Nasdaq's Behind the Bell with uniQure CEO Matt Kapusta
มุมมอง 4155 ปีที่แล้ว
Our CEO Matt Kapusta spoke with Nasdaq's Behind The Bell after ringing the Opening Bell to talk about about what sets uniQure apart and how the company is working to revolutionize the treatment of genetic diseases through gene therapy.
How AAV Gene Transfer Works - General Audience
มุมมอง 88K7 ปีที่แล้ว
This brief animation, designed for a general audience, illustrates the basics of AAV gene transfer technology.
How AAV Gene Transfer Works - Expert Audience
มุมมอง 37K7 ปีที่แล้ว
This brief animation, designed for medical and scientific audiences, illustrates the basics of AAV gene transfer technology.
uniQure CEO Matt Kapusta
มุมมอง 5867 ปีที่แล้ว
uniQure's CEO Matt Kapusta discusses gene therapy's potential to revolutionize the healthcare industry, and uniQure's mission to deliver transformative gene therapy products to patients with serious unmet medical needs.
The most beautiful video I have ever seen on this topic. Such a piece of art!!! 💌💌💌💌💌
Johnson Kimberly Wilson Jessica Davis Jason
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).
Could you make this contagious? like a disease that forces peoples genes to change?
Amazing how life is made
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.
I don't understand. Please elaborate on that. Why do you think integrating the foreign DNA into the host genome would be beneficial?
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
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.
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?
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.
Should I trust medical product like this cause I've went without all of it and I seem to be fine but idk
obviously, you'd only need this if you're sick
If you don't have genetic pathology, why do you want to use this kind of treatment ?
This is a work of art 😍❤
Please post video updates
can gene therapy be used to increse Neurogenesis ?
the next 100 years is going to be amazing .. don't buy pills ,, just edit your genes
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.
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
the animations here are insane! Thank you, very useful to see the whole process animated like this, very educational. helps understand.
What are the latest developments in the drug statement for Huntington's patients?
What are the latest developments in the drug statement for Huntington's patients?
can you cite the research you're summarizing?
I am waiting for a great news ।
Are there any updates on AMT-150 initiation of IND?
You guys are about to save my life and the life of so many others. Keep up the good work!
Does anyone know what company produced this animation?!
Hey Guys 😊 I'm giving away $250 coupon for Apple vs Samsung Challenge... Those who are interested might want to take a closer look to this 👉 bit.ly/Apple-Vs-Samsung-Challenge #samsungvsiphonecamera, #applevssamsungcomparisonchart, #iphonevssamsungwhichisbetter2019, #shouldigetaniphoneorsamsung, #appleorsamsungpoll, #applevssamsungsales2020, #applevssamsungpricecomparison, #applevssamsungcompanycomparison, #usa, #unitedstates, #us 今後は気をライブ配信の再編ありがとうです！この日のライブ配信は、かならりやばかったですね！１万人を超える人が見ていたもん（笑）やっぱり人参最高！まさかのカメラ切り忘れでやら1かしたのもドキドキでした -String.Spintax- . 🤩 在整個人類歷史上，強者，富人和具有狡猾特質的人捕食部落，氏族，城鎮，城市和鄉村中的弱者，無`'守和貧窮成%員。然而，人類的生存意願迫使那些被拒絕，被剝奪或摧毀的基本需求的人們找到了一種生活方式，並繼續將其DNA融入不斷發展的人類社會。 說到食物，不要以為那些被拒絕的人只吃垃圾。相反，他們學會了在被忽視的肉類和蔬菜中尋找營養。他們學會了清潔，切塊，調味和慢燉慢燉的野菜和肉類，在食品市場上被忽略的部分家用蔬菜和肉類，並且學會了使用芳香的木煙 （如山核桃 😍
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.
We are trying our best! <3
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 ...
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).
what an amazing animation!!!!
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
When will this treatment be available for people who suffer from this horrific disease?
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.
@@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.
@@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.
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.
You got here because of Bill Gates Ted talk?
No, but I was definitely thinking about him while watching.
No, i'm studying for my endsemester exams as a first yearite in college
thanks
Great video! Very well portrayed.
Smart animation.
thats the beauty of molecular biology...i am in love ...😍😍
this is LIIT
Magnificent video. Thank you UniQure!!
Animation😮