🧬 Faced with the challenge to reliably synthesize and produce complex plasmid DNAs used in the production of viral vectors for gene therapy, Novel Biotechnology submitted 51 UTR sequences for complexity screening to three commonly used DNA synthesis vendors, as well as to Ansa. As they reported in this poster, the majority of the sequences were rejected outright by most vendors, but 100% were accepted for synthesis by Ansa. "Here we have shown how innovations in DNA synthesis and assembly enable the routine production of plasmid designs that would have been difficult, if not impossible, to generate using conventional techniques. These innovations include the use of Ansa’s enzymatic DNA synthesis platform in conjunction with Novel Bio’s in-house workflows for plasmid DNA production. The use of these technologies enabled us to seamlessly deliver on a complicated gene therapy project. Without this capability, this project would have become considerably more complex and costly." Thanks for sharing this success story, Novel Biotechnology Inc.! Interested in putting our #complexDNA synthesis services to the test on your project? Learn how to join our Early Access Program: https://lnkd.in/grMhxgfN #DNAsynthesis #longDNA #Ansamers #biotech #synbio #SyntheticDNA #DNAInnovation #complexDNA #customDNA #AnsamerAnswers
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#AAVdownstream challenges: expert insights #AAVvectors are a primary delivery mechanism for #genetherapies due to low immunogenicity, long lasting gene expression, and the ability to target specific tissues. The field of #AAV manufacturing is rapidly expanding, but there are challenges in the downstream processing of these #viralvectors. Next month, an expert panel including Nathalie Clément (VP, Vector Development for Translational Gene Therapies, Siren Biotechnology), Matt Roach (Director of AAV Production, BridgeBio), Nicolas Laroudie (Senior Field Application Scientist, Thermo Fisher Scientific), and William Kish (Principal Scientist - Downstream Process Sciences Engineering, Taysha Gene Therapies) will provide an opportunity to learn more about the latest trends and issues in #AAVmanufacturing, and specifically, how to address the most common downstream processing challenges Register for this #webinar below:
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Today is National DNA Day, a day to celebrate the groundbreaking discovery of the double helix in 1953 and the successful completion of the Human Genome Project in 2003. On days like today, we are reminded of the important research and discovery that have resulted in two of the greatest scientific advancements of all time, both of which have accelerated advancements in #GeneTherapy and remain a significant driver of progress and #innovation to date. At Purespring, we have built on these powerful discoveries and are proud to be the first company to treat kidney diseases by directly targeting the podocyte with AAV gene therapy. Learn more about our novel approach here: https://lnkd.in/d8_G__CR #KidneyDisease #DNADay #Biotech #CellAndGeneTherapy
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🚀 Cyclodextrins: New Horizons in Gene Delivery 🧬 In gene therapy, safety and efficiency are paramount. Cyclodextrins, cyclic oligosaccharides derived from starch, are emerging as promising non-viral nanocarriers for genetic material. 🔬 Key Benefits: Biocompatibility: Low toxicity, minimal adverse reactions. Versatility: Efficiently carries DNA, RNA, and siRNA. Controlled Release: Ensures targeted, prolonged therapeutic effects. 🚧 Challenges: Improving transfection efficiency and in vivo performance remains a focus. 🔍 GENEGUT Project: Our Horizon 2020 grant project aims to develop tools for delivering oligonucleotides to treat Crohn's disease. Cyclodextrins offer a safer, versatile alternative to viral vectors, potentially revolutionizing gene therapy. #GeneTherapy #Cyclodextrins #Biotech #Healthcare #GENEGUT
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🔬 Research Alert! published in #DrugDevIndPharm 2022, VOL. 48, NO. 1, 1–11 🌟 A cationic cyclodextrin derivative-lipid hybrid nanoparticles for gene delivery effectively promotes stability and transfection efficiency 🌟 This research article explores the development of a novel gene delivery system using lipid-polymer hybrid nanoparticles (LHNPs) composed of α-cyclodextrin-conjugated generation-2 polyamidoamine dendrimers (CDG2) and lipids. The study demonstrates that these CDG2-LHNPs exhibit superior stability and transfection efficiency compared to traditional liposomes and polymers. The nanoparticles were stable for up to 15 days at various temperatures and showed high transfection efficiency mediated by multiple endocytosis pathways, particularly caveolae-mediated endocytosis, which helps avoid lysosomal degradation. The hybrid system's enhanced cell uptake and proton buffering capacity contribute to its effectiveness, making it a promising candidate for gene therapy applications. 📖 Read the full article here: https://lnkd.in/gcxwaYrs #GeneTherapy #Nanoparticles #DrugDelivery
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Pursuing Bsc Honours in Biotechnology | Final Year Student | Exploring the Frontiers of Biotech Innovation | Passionate about Innovating Healthcare | Biotech Enthusiast 🔬🧬
◽️𝐇𝐮𝐦𝐚𝐧 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐓𝐫𝐢𝐚𝐥𝐬: 🔹Human gene therapy trials involve experimental treatments where defective genes in a person's cells are corrected or replaced. 🔹These trials aim to treat genetic disorders by introducing healthy genes into the patient's cells, either in vivo (directly into the body) or ex vivo (cells modified outside the body and then reintroduced). 🔹The process includes preclinical research, clinical trials with phases to test safety and efficacy, and stringent regulatory oversight to ensure patient safety. 🔹Successful trials can lead to groundbreaking treatments for conditions previously deemed untreatable. Image source: Biotechnology by U.Satyanarayana #biotechnology #biotechnologystudent #biotech #science #research #genetics #sciencecommunity #lifescience #carrerinbiotech #futureofbiotech #genetherapy #dikshitaramse
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Let's learn about CRISPR gene editing !! CRISPR gene editing is a revolutionary technology that allows scientists to precisely alter DNA within living organisms. Using a specialized enzyme called Cas9, guided by a short RNA sequence, CRISPR can target specific genes and either modify, delete, or replace them. This technology has transformed genetic research, enabling the correction of genetic defects, the study of gene functions, and the development of therapies for a wide range of diseases. Its precision, efficiency, and versatility make CRISPR a powerful tool in advancing personalized medicine and biotechnology. Follow Krithina - Trainer@Atheenapandian for more updates CRISPR Therapeutics CRISPR QC CRISPR genome editing by IDT @crispr
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🌟 The Future of Gene Therapy: Why LNPs Are Gaining Momentum! 🌟 Traditional gene therapy often relies on viral vectors, which can pose safety risks, cause immune reactions, and have limited capacity for genetic material. These challenges have restricted their broader clinical use. Enter Lipid Nanoparticles (LNPs)! 🎉 These non-viral vectors are changing the game with their exceptional safety profile, ability to administer multiple doses, and large genetic capacity. Plus, they’re easier to design and manufacture compared to their viral counterparts. LNPs are quickly becoming the preferred choice in gene therapy, offering a promising alternative that overcomes many of the limitations of traditional methods. 🚀💡 #GeneTherapy #Innovation #LipidNanoparticles #Healthcare #Biotechnology #NonViralVectors https://lnkd.in/gpQynpzH
Lipid Nanoparticles: Revolutionizing Gene Therapy and Vaccines
us.huatengsci.com
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Driving Access to Health |Ex Head-AI platforms |Serial Innovator| Independent Director|Purpose Alchemist
The Gene light switch Today's gene therapy faces a critical challenge: traditional methods introduce foreign proteins to regulate genes, often provoking detrimental immune responses as the body combats these alien elements. Addressing this, Baylor College of Medicine researchers, led by Dr. Laising Yen, have developed an innovative RNA-based technology. Their method employs small, FDA-approved molecules to interact with RNA, avoiding immune activation. This 'light switch' for genes, adjustable with common drugs like tetracycline, promises precise control within safe drug concentrations. This breakthrough, a decade in the making and published in Nature Biotechnology, has potential applications in various conditions, including genetic disorders and cancer, offering a more targeted and safer approach to gene therapy. Amit Saxena Ajay Nandgaonkar Sanju Senthil Kumar Suchitaa Paatil Dr Taruna Anand #accessAIchemy #access #genetherapy read more :https://lnkd.in/ggbJ_DAm
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The surge in groundbreaking research and medical advancements is propelling the cell and gene therapy sector to unprecedented heights. Some examples include Synthetic biology, In vivo CAR-T cell engineering, iPSC-derived therapies, and many more. Want to see the complete list? Our whitepaper spotlights emerging cell and gene therapy technologies that can shape the biotech industry for the next decade. Dive in to learn about the challenges and opportunities that lie ahead. https://lnkd.in/eiAu9rjD
Preparing for the Future: Emerging Technologies Shaping the Cell & Gene Therapy Landscape - Alliance for Regenerative Medicine
https://alliancerm.org
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Don't forget to join us today at 3pm GMT / 4pm CET /10am EST for the first webinar in our 3 part series on CRISPR and Gene Editing ONLINE. If you can't make it you can still register to catch up on-demand: https://lnkd.in/dA9KycdX Today, we'll be discussing advances in gene editing systems. We'll be highlighting their applications and benefits and discussing the future of gene editing technologies, both from a technological standpoint and in relation to future therapeutics. Talk 1: Precise Transcript Targeting by CRISPR-Csm Complexes David Colognori, Post-Doctoral Associate, Doudna Lab, UC Berkeley Talk 2: PRCISR CRISPR: Vivlion’s CRISPR-enabled PCR-free discovery platform Martin Wegner, Head of R&D, Vivlion GmbH Talk 3: Precise and Versatile Genome Engineering with Prime Editing Andrew Anzalone, Head of the Prime Editing Platform and Scientific Co-Founder, Prime Medicine, Inc. #CRISPR #geneediting
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Principal Scientist - Nucleotide Chemistry at Miltenyi Biotec
3moCongratulations