GenScript USA Inc.
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Advancing Gene Editing Technologies with Spherical Nucleic Acids
Dr. Sergej Kudruk
International Institute for Nanotechnology (IIN) Postdoctoral Fellow, Northwestern University, Department of Chemistry
Nanomaterials have already begun to play a pivotal role in overcoming the limitations of traditional drugs by enhancing drug delivery, improving bioavailability, overcoming biological barriers in the treatment and prevention of diseases. Spherical Nucleic Acids (SNAs) stand out as a versatile class of nanomaterials that have been utilized in applications spanning diagnostics and therapeutics. SNAs, comprised of a nanoparticle core densely functionalized with radially oriented oligonucleotides, offer unique advantages for nanomedicine. Their 3-dimensional structure renders SNAs with superior properties in comparison to linear nucleic acid molecules, including enhanced cellular uptake, resistance to nuclease degradation, and superior target affinity. These qualities make SNAs ideally suited for their application towards effective gene editing technologies. This presentation will cover two recent examples of employing SNAs in gene editing paradigms. First, an approach that enhances the therapeutic potential of small interfering RNAs (siRNAs) when formulated as SNAs is described. Until now, the gene silencing activity of SNAs had been hindered by endosomal entrapment. Here, a simple yet innovative strategy that employs calcium chloride (CaCl2) salting in SNA formulation results in enhanced cytosolic delivery and gene silencing activity by up to 20-fold across diverse cell lines, indicating a promising avenue for overcoming existing limitations. In a second, related story, SNAs are employed for the purposes of performing CRISPR/Cas9 systems for genome editing. In this work, Cas9 protein-cored SNAs, densely modified with DNA and preloaded with single-guide RNA, exhibit enhanced cellular uptake and stability, achieving notable genome editing efficiencies across multiple cell lines. The integration of GALA peptides and nuclear localization signals further enhances endosomal escape and nuclear localization. Taken together, this work underscores the importance of precise control over molecular interactions at the nanoscale and the potential of SNA-based nanotechnologies for gene editing applications.
Learning Objectives:
- Spherical Nucleic Acids (SNAs), exhibit properties making them ideal constructs for nanomedicine and effective gene editing technologies. This talk describes routes of employing SNAs to markedly improve the cytosolic delivery of siRNAs and enhanced genome editing and delivery of CRISPR/Cas9 platforms.
มุมมอง: 79

วีดีโอ

Autoimmune Diseases, the Arising Opportunities for Cell Therapies
มุมมอง 41หลายเดือนก่อน
Dr. Dong Geng VP, Head of Early-stage Drug Development Legend Biotech CAR-T cell therapies are now the established treatments for patients with relapsed and/or refractory B cell lymphomas, B cell acute lymphoblastic leukemia, and multiple myeloma. Through deep depletion of targeted malignancy cells, CD 19-targeted and BCMA-target CAR-T treatment could lead to long-term remissions, in some cases...
Synthetic biology approaches to improve cell therapy function
มุมมอง 63หลายเดือนก่อน
Dr. Michelle Hung Principal Scientist GeneFab There has been an explosion in the number of genetic medicines, including, cell & gene therapies in clinical development. These therapies contain thousands to billions more atoms than small molecules and biological drugs. Thus the design and manufacturing of these advanced therapies is much more complex than more established therapeutic modalities. ...
The Power of Automation: How Robotics Can Dramatically Reduce Cell Therapy Manufacturing Costs
มุมมอง 96หลายเดือนก่อน
Dr. Fred Parietti Co-founder and CEO of Multiply Labs There’s an elephant in the room when it comes to cell therapy. Hundreds of thousands of patients are in need of these life saving therapies - but the vast majority can’t access the treatment. This is because cell therapy manufacturing is reliant on a highly-skilled and limited labor pool, which results in prohibitively high prices and lack o...
Programmable Genomic Integration: a technology that enables the integration of large DNA sequences
มุมมอง 78หลายเดือนก่อน
Dr. Sandeep Kumar Senior Director, Platform Tome Biosciences Tome Biosciences is leading the field of programmable genomic integration (PGI). Building on founding technologies obtained from MIT and Replace Therapeutics, Tome has developed and refined PGI resulting in stable integration of multiple kilobases of DNA at user-defined locations. Integrase-mediated PGI (I-PGI) uses a Cas9 nickase wit...
Cytokine Armored CAR NK Cell Therapy for Advanced Ovarian Cancer
มุมมอง 51หลายเดือนก่อน
Dr. Branden Moriarity Associate Professor, Medical School, University of Minnesota Immunotherapy using chimeric antigen receptor (CAR) expressing NK cells engineered via viral vectors has shown exceptional efficacy against hematologic cancers in clinical trials. This method, however, is limited in cargo capacity and large-scale manufacturing for clinical use is cost-prohibitive to most academic...
De novo gene synthesis by an antiviral reverse transcriptase
มุมมอง 186หลายเดือนก่อน
Dr. Samuel Sternberg Associate Professor, Department of Biochemistry and Molecular Biophysics, Columbia University Bacteria defend themselves from viral infection using diverse immune systems, many of which sense and target foreign nucleic acids. Defense-associated reverse transcriptase (DRT) systems provide an intriguing counterpoint to this immune strategy by instead leveraging DNA synthesis,...
4th Annual Gene & Cell Engineering Virtual Summit Recap
มุมมอง 53หลายเดือนก่อน
Watch our recap of the event! Did you miss it or weren't able to attend the entire event? No worries! You can still register now and watch on demand: bit.ly/4cPC9f6
Accelerating Cell & Gene Therapy Success with cGMP Gene Editing Components and Regulatory Compliance
มุมมอง 1542 หลายเดือนก่อน
The manufacturing of gene editing (GE) components is a pivotal aspect of GE-based Cell and Gene Therapy development, requiring meticulous attention to product quality and regulatory compliance throughout the development lifecycle. In this webinar, we will discuss the key differences between GE components used from early discovery through to clinical application, and provide insight into how pha...
AI based Antibody Therapeutics Developability Assessment and Its Engineering
มุมมอง 2073 หลายเดือนก่อน
Learn from an informative webinar about how AI-based platforms can facilitate the development process for antibody therapeutics and ensure candidates are not only therapeutically effective but also viable for large-scale production and clinical use. Monoclonal antibodies (mAbs) have become essential in treating a wide spectrum of diseases, with over 100 therapeutic antibody products now approve...
AET London 2024: Bispecific Antibody Therapeutics: Challenges, Opportunities and Future Directions
มุมมอง 1293 หลายเดือนก่อน
This talk, “Bispecific Antibody Therapeutics: Challenges, Opportunities and Future Directions” was given during the 2024 Antibodies Engineering & Therapeutics Europe meeting in London, UK, by Robert Ford, PhD, Field Application Specialist at GenScript. In this presentation, you will learn about bi-specific antibodies (bsAbs) demonstrating novel functionalities that yield remarkable promise in i...
Streamlining Cell and Ex Vivo Gene Therapy Manufacturing Innovative Solutions from GenScript
มุมมอง 1445 หลายเดือนก่อน
Cell and gene therapies are on the brink of transforming the pharmaceutical industry, presenting unique challenges such as ensuring the quality and consistent supply of reagents. Reagents play a crucial role in various stages of cell therapy development, from cell isolation and expansion to gene editing and characterization. Any inconsistency or variation in the quality of reagents can affect t...
Complete Toolbox for Gene Therapy Development with Live Q&A
มุมมอง 1065 หลายเดือนก่อน
Gene therapy has revolutionized the field of medicine, and it has the potential to cure a wide variety of genetic disorders. Gene therapy involves the introduction of a functional gene into the patient's cells to replace the missing or defective one. The two main technologies used for gene therapy are viral-based and non-viral gene editing. The primary advantage of viral-based gene editing is t...
Advanced Solutions for mRNA Vaccine Development with Live Q&A
มุมมอง 1115 หลายเดือนก่อน
mRNA vaccines represent a pivotal innovation in prophylactic healthcare, offering a rapid response to emerging infectious diseases. Leveraging the cellular machinery to produce antigenic proteins, these vaccines induce robust immune responses without introducing live pathogens. Recent developments have focused on optimizing mRNA stability, delivery systems, and targeted immune activation. This ...
Bispecific Antibody Overview & Production with CrossMab Technology
มุมมอง 7445 หลายเดือนก่อน
In this webinar, Dr. Sai Wang, protein senior scientist at GenScript Biotech Singapore, will discuss the advantages of bispecific antibodies (bsAbs), their mechanism of action (MOA), and the development history of bsAb platform. She will also help attendees to gain an understanding of the CrossMab production method and its significance in the field of antibody engineering. Additionally, Dr. Wan...
Advancing Brain Shuttle-Enabled Therapeutics for Efficient Delivery to CNS
มุมมอง 2096 หลายเดือนก่อน
Advancing Brain Shuttle-Enabled Therapeutics for Efficient Delivery to CNS
Life Science Research Grant Program
มุมมอง 777 หลายเดือนก่อน
Life Science Research Grant Program
Enhancing Treatments using Targeted Protein Degradation
มุมมอง 2248 หลายเดือนก่อน
Enhancing Treatments using Targeted Protein Degradation
The Future of Plasmid Maxi-prep is Here: AmMag™ Quatro Automated Solution
มุมมอง 1928 หลายเดือนก่อน
The Future of Plasmid Maxi-prep is Here: AmMag™ Quatro Automated Solution
Robust production of antibody fragments and their therapeutic applications
มุมมอง 1878 หลายเดือนก่อน
Robust production of antibody fragments and their therapeutic applications
PEGS Europe 2023: Empowering Therapeutic Antibody Development
มุมมอง 859 หลายเดือนก่อน
PEGS Europe 2023: Empowering Therapeutic Antibody Development
Bispecific Antibodies Structural Design, Production and Purification Strategies
มุมมอง 1.3K9 หลายเดือนก่อน
Bispecific Antibodies Structural Design, Production and Purification Strategies
Best in class antiviral antibodies from cognate recombinant antibody repertoires of human donors
มุมมอง 529 หลายเดือนก่อน
Best in class antiviral antibodies from cognate recombinant antibody repertoires of human donors
GenScript x XTalks Webinar: Adaptive Immune Response in Narcolepsy Type 1
มุมมอง 10011 หลายเดือนก่อน
GenScript x XTalks Webinar: Adaptive Immune Response in Narcolepsy Type 1
What to Expect from a CRO
มุมมอง 12811 หลายเดือนก่อน
What to Expect from a CRO
What to Expect from a CRO: What can you expect from GenScript once a project starts?
มุมมอง 5311 หลายเดือนก่อน
What to Expect from a CRO: What can you expect from GenScript once a project starts?
What to Expect from a CRO: How to establish a Contract for Services
มุมมอง 3011 หลายเดือนก่อน
What to Expect from a CRO: How to establish a Contract for Services
What to Expect from a CRO: How to start your Partnership with GenScript
มุมมอง 3011 หลายเดือนก่อน
What to Expect from a CRO: How to start your Partnership with GenScript
What to Expect from a CRO: Why start your partnership with GenScript?
มุมมอง 2211 หลายเดือนก่อน
What to Expect from a CRO: Why start your partnership with GenScript?
What to Expect from a CRO: The GenScript Experience
มุมมอง 5411 หลายเดือนก่อน
What to Expect from a CRO: The GenScript Experience

ความคิดเห็น

  • @ericamadobegines8764
    @ericamadobegines8764 12 ชั่วโมงที่ผ่านมา

    That is awesome ❤. Great great job. Currently I work with polycistronic genes that are very much in the limit of the cargo that AAV accepts, 4.8-5 kbp, making it very challenging, it would be great that Genscript develop strategies for this kind of cargo, larger than 4.5 kbp without the need to resort to infect with complementary plasmids to work in trans

  • @cassandratorres1737
    @cassandratorres1737 11 วันที่ผ่านมา

    I pray a world health organization will give these children and people with eb the necessities they need to live comfortably 🙏🏽💗 Because these diseases affect small amounts of people it isn't acknowledged. BUT in a world with BILLIONS of people this "small group" is affecting hundreds of thousands of innocent babies and small children. I hope awareness and financial support reach these organizations in time to make a difference 💗🙏🏽💗🙏🏽

  • @LawrenceJennifer-x8n
    @LawrenceJennifer-x8n 23 วันที่ผ่านมา

    I'm happy for recommend DOC UROMI on TH-cam to everyone, as he cure my STD.

  • @deborahloe3028
    @deborahloe3028 26 วันที่ผ่านมา

    Praying for new donors and creative miracles. In these babies bodies

  • @hherrera9855
    @hherrera9855 หลายเดือนก่อน

    Please consider fixing the issues that garners this organization with such a low charity rating on "Charity Navigator". I would consider it a privilege to donate but must also be a good steward of my resources and ensure they are being used for the intended purpose. Thank you!

  • @IOSARBX
    @IOSARBX หลายเดือนก่อน

    GenScript USA Inc., awesome video it was really entertaining

  • @shangsphere
    @shangsphere 3 หลายเดือนก่อน

    This is a very nice introduction. Thank you!

  • @PreciousGda
    @PreciousGda 4 หลายเดือนก่อน

    It's a great privilege coming across your TH-cam channel Dr Odija. You have always been there for me health wise. I can vividly remember how you turned up for me when I was down with HIV virus. Your contribution and care was highly appreciated. You are the best doctor I have ever come across. Thank you so much for the difference you make in the lives of your patients! Your kindness, sincere caring, and concern make everything better and are a great encouragement ,,.

  • @MurugeshMurugesh-sk3sj
    @MurugeshMurugesh-sk3sj 4 หลายเดือนก่อน

    How to M13 vector construct using laptop

  • @VictoriaNskn
    @VictoriaNskn 4 หลายเดือนก่อน

    am definitely happy to have met Dr Odija on TH-cam channel. You have done a great improvement in my life. Thanks for curing my HIV completely. May God continue to bless Dr Odija

  • @angelthman1659
    @angelthman1659 4 หลายเดือนก่อน

    Blah, blah, blah, blah. Now translate into English.

  • @ladyalexander2003
    @ladyalexander2003 4 หลายเดือนก่อน

    Immune response does not imply immunity! If someone is poisoned with heavy metals or other toxin any bacterial infection etc enters their body they get an immune response!

  • @drummond06
    @drummond06 5 หลายเดือนก่อน

    This video is giving "Turbo Encabulator"

  • @billyt9921
    @billyt9921 5 หลายเดือนก่อน

    Doing the devils work

  • @abdelmoujoudfaris1162
    @abdelmoujoudfaris1162 5 หลายเดือนก่อน

    Thank you

  • @sebastianurbanski8072
    @sebastianurbanski8072 5 หลายเดือนก่อน

    dulce

  • @chattafadilat
    @chattafadilat 5 หลายเดือนก่อน

    I found hope and my complete treatment for my Infection through Dr Ogoh Herbal Medication. ...

  • @Shyguy71588
    @Shyguy71588 6 หลายเดือนก่อน

    Only God can make such complexity such as cells, organs and organisms. We are this far into technology yet can barely make cells or living things with deoxyribonucleic acid.

  • @cherrymarriedindiscord1404
    @cherrymarriedindiscord1404 6 หลายเดือนก่อน

    All my respect for Dr. Liu Pel but it was really hard to follow the ideas she was presenting

  • @jldonnell1
    @jldonnell1 6 หลายเดือนก่อน

    Where can the rest of this presentation be found?

  • @AmandaJon-nc9qw
    @AmandaJon-nc9qw 7 หลายเดือนก่อน

    😊 All thanks to Dr abiola for curing me permanently from HIV #drabiola

  • @dineshmishra6538
    @dineshmishra6538 8 หลายเดือนก่อน

    Very nice information

  • @dineshmishra6538
    @dineshmishra6538 8 หลายเดือนก่อน

    Can I get certificate

  • @Ddd-ij7ct
    @Ddd-ij7ct 8 หลายเดือนก่อน

    I’m so happy for DR OGOH on TH-cam for his good HIV MEDICATION that defeated my virus.

  • @Ddd-ij7ct
    @Ddd-ij7ct 8 หลายเดือนก่อน

    I’m so happy for DR OGOH on TH-cam for his good HIV MEDICATION that defeated my virus. 0:03 😊

  • @bbbbb25
    @bbbbb25 8 หลายเดือนก่อน

    wtf is she saying bruh 💀

  • @ladyalexander2003
    @ladyalexander2003 10 หลายเดือนก่อน

    Except DNA is far more than Guanine Adenine cytosine & thyamine maybe learn how DNA actually works and the information it stored and stop oversimplifying the human body and Gods work you don’t understand properly and you’ll realise your causing problems not solving them at all this is just idiotic foolery playing with peoples lives and causing disease! Not curing it!

  • @craig4android
    @craig4android 10 หลายเดือนก่อน

    what is a fusion tag?

  • @noreenbashir3936
    @noreenbashir3936 10 หลายเดือนก่อน

    Can we use the baculovirus system for vaccine for poultry or it only go with the mammalian cells

  • @SeanLawlorNelson
    @SeanLawlorNelson 10 หลายเดือนก่อน

    This is critically important science I too have long been working on, more recently with Mr. Elon Musk in direct live teleconferencing through the modality of XAI. From my view, one of the more important applications of this science will be to proceed with our creation of physical, industrial, etc., droid workers with synthesized DNA; This allows them to compete and be eliminated, thus becoming fitter, more adapted workers and also thinking machines; but most importantly, it gives them the Darwinian and Lamarckian motivation that makes humans so resourceful and adaptive. I have always known that despite some fireworks, AI would be limited, really only semi-'intelligent' until it had the genetic drive to survive, excel, thrive, and reproduce potentially in plethora of progeny. The potential is enough as long as it's real. These droid workers will be alert, linguistic, and self-maintaining like human workers almost; and will have certain applications. It is not my intention to replace the human work force. I greatly admire and often cite as a model Phetchaburi Rajabhat University where I taught and employs so many, all of 100 street-sweeping specialists with a couple rakes and a bag; this is one part of university maintenance and it employs many and makes for a great community. And for those who do not work, perhaps they create art or go to church or temple as laity; They should eat, too. I am a socialist; do not misunderstand nor abuse my system please. We create and share abundance in civilized manner.

  • @agathabukowski7941
    @agathabukowski7941 10 หลายเดือนก่อน

    The great Dr Kanayo Peterson saved my life when I discovered his TH-cam page and contacted him through WhatsApp about how badly I was struggling with my hiv aids infection. He sent me his wonder tonic which saved my life.

  • @Earthoceanscast
    @Earthoceanscast 11 หลายเดือนก่อน

    How is possible the government don't provide this kids with everything they need ?

  • @ralphstrong3117
    @ralphstrong3117 11 หลายเดือนก่อน

    'PromoSM'

  • @newveal6593
    @newveal6593 ปีที่แล้ว

    “Come On” by Doc Hartley is truly one of the greatest musical masterpieces of our era

  • @yushzh8369
    @yushzh8369 ปีที่แล้ว

  • @goldenantenterprizes
    @goldenantenterprizes ปีที่แล้ว

    This woman knows nothing about immunology and is spouting rubbish in poor English.

  • @azizehshadi7605
    @azizehshadi7605 ปีที่แล้ว

    ❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤❤

  • @charlikaushal9003
    @charlikaushal9003 ปีที่แล้ว

    Please share the link for registration.

  • @73fearnot
    @73fearnot ปีที่แล้ว

    I would like to know a bit more about how the gene therapies for baby skin blistering disease are being studied and produced. Are they ethically researched? Are they studied and/or propagated using aborted human cell lines? I found out way more than I ever wanted to know concerning many other pharmaceutical interventions.

  • @jimmelton3216
    @jimmelton3216 ปีที่แล้ว

    Dr Ejemhen on has the cure for hiv…

  • @amosmak
    @amosmak ปีที่แล้ว

    Nice work, Jane!

  • @uuubeut
    @uuubeut ปีที่แล้ว

    When monsters become GOD

  • @sahandkh2458
    @sahandkh2458 ปีที่แล้ว

    thanks

  • @0824mnkfjxb
    @0824mnkfjxb ปีที่แล้ว

    All the love to you for helping so many children ❤❤❤❤❤❤

  • @mdjunad5771
    @mdjunad5771 ปีที่แล้ว

    How is lily now?

  • @goncalofigueiredo5986
    @goncalofigueiredo5986 ปีที่แล้ว

    Could you just clarify me in 27:36. When you say n-terminus and c-terminus in the MCS, how could that be possible? Don't they only exist in protein chains? How can the tags be put in n-terminus or c-terminus? Do you put them before translation? But then how do they bind to the resultant protein? So these proteases are a post-translational treatment, how do you insert them into the host? Plasmids?

  • @Aoirsae
    @Aoirsae ปีที่แล้ว

    great

  • @alejandropikoulasplata5524
    @alejandropikoulasplata5524 ปีที่แล้ว

    Thank you so much!!

  • @InquilineKea
    @InquilineKea ปีที่แล้ว

    omg how about targeted delivery of things that enhance asymmetrical segregation of stem cell division so they don't deplete with age?

  • @InquilineKea
    @InquilineKea ปีที่แล้ว

    Ok can we use this to deliver intelligence genes into the brain b/c it's impt for AI alignment