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Showing 1 to 16 of 16 for “"mRNA delivery"”.

  1. Optimization and analysis of lipid nanoparticles for in vivo mRNA delivery

    Messenger RNA (mRNA) therapeutics have the potential to treat a diverse array of diseases requiring protein expression, with applications in protein replacement therapies, immunotherapies, and genome engineering. However, the intracellular delivery of mRNA is challenging and necessitates a safe and …

    mit Repository record for Optimization and analysis of lipid nanoparticles for in vivo mRNA delivery (opens in a new tab)

  2. Development of polymer - lipid nanoparticles for potent mRNA delivery to the lung

    Messenger RNAs (mRNAs) are an emerging therapeutic modality that holds great promise to specifically and completely treat genetic disease. mRNA has been used as a vaccine, as a protein replacement therapy, and even as a means of inducing permanent genomic editing via CRISPR. However, unlike …

    mit Repository record for Development of polymer - lipid nanoparticles for potent mRNA delivery to the lung (opens in a new tab)

  3. Targeting and Reprogramming Tumor-Associated Macrophages with Polymeric Nanoparticle-Mediated mRNA Delivery for Cancer Treatment

    … is in vivo reprogramming using messenger RNA (mRNA). mRNA can directly encode therapeutically proteins without genomic integration, avoiding the risk of insertional mutagenesis. Additionally, direct programming in vivo offers a more cost-effective and quicker alternative. Since mRNA is …

    washington Repository record for Targeting and Reprogramming Tumor-Associated Macrophages with Polymeric Nanoparticle-Mediated mRNA Delivery for Cancer Treatment (opens in a new tab)

  4. Development and applications of peptide barcoded nanoparticles for high-throughput screening of mRNA delivery materials in vivo

    mRNA lipid nanoparticles (LNP) present a promising class of therapeutics, with broad applications in protein replacement therapy, gene editing, immunotherapy, and vaccines, owing to their versatility and precise nature. While recent years have seen dramatic improvements in the safety and efficacy …

    mit Repository record for Development and applications of peptide barcoded nanoparticles for high-throughput screening of mRNA delivery materials in vivo (opens in a new tab)

  5. Design and synthesis of ionisable amino-polyester lipid nanoparticles for extrahepatic RNA delivery

    Messenger RNA (mRNA)-based therapeutics represent a powerful platform with the potential to transform modern medicine by targeting diseases at their molecular origins. Advances in mRNA chemistry and delivery technologies have enabled applications ranging from vaccines and cancer immunotherapy to …

    cork Repository record for Design and synthesis of ionisable amino-polyester lipid nanoparticles for extrahepatic RNA delivery (opens in a new tab)

  6. FMRP dependent synaptic delivery of messenger RNA

    … the regulatory role of FMRP involved in synaptic mRNA delivery. In chapter 1, the molecular mechanism of local protein synthesis and synaptic mRNA delivery and its roles in Fragile X syndrome and learning and memory are introduced. In chapter 2, my data demonstrated that FMRP can facilitate mRNA

    uiuc Repository record for FMRP dependent synaptic delivery of messenger RNA (opens in a new tab)

  7. Tie-ing the endothelium: development of an mRNA-based nanotherapeutic to restore microvascular barrier function for the treatment of sepsis-induced multiple organ failure

    … vaccines has showcased the use of messenger RNA (mRNA) as a therapeutic modality for transient upregulation of protein expression and precise modulation of cellular pathways. However, intracellular delivery of mRNA remains a hurdle due to its large size, polyanionic nature, and susceptibility to …

    cork Repository record for Tie-ing the endothelium: development of an mRNA-based nanotherapeutic to restore microvascular barrier function for the treatment of sepsis-induced multiple organ failure (opens in a new tab)

  8. Design, synthesis, and biological evaluation of diketopiperazine based ionizable lipids for the in vivo delivery of messenger RNA

    … of specific proteins. The successful delivery of messenger RNA (mRNA) to target cells in the body could accomplish this goal, but serious limitations with its systemic delivery must still be overcome. Recently, lipid nanoparticles (LNPs) have shown promise for mRNA delivery in vivo, …

    mit Repository record for Design, synthesis, and biological evaluation of diketopiperazine based ionizable lipids for the in vivo delivery of messenger RNA (opens in a new tab)

  9. Development and mechanistic analysis of in vivo liposomal nanoparticle delivery of siRNA and mRNA

    A limited number of siRNA and mRNA based therapeutics have been developed largely due to the difficulties of efficaciously delivering RNA to cells in vivo. Liposomal nanoparticles (LNPs) have shown some success, but their design is limited by both a lack of information concerning the optimal lipid …

    mit Repository record for Development and mechanistic analysis of in vivo liposomal nanoparticle delivery of siRNA and mRNA (opens in a new tab)

  10. Elucidating the Impact of Endogenous Proteins on Lipid Nanoparticle Organ-Targeting Outcomes

    … the cytosol, where it is biologically active, a delivery system is necessary to circumnavigate these obstacles and ensure targeting of the proper cell type(s). Presently, lipid nanoparticles (LNPs) represent the most clinically-advanced system for delivery of multiple classes of RNA, including …

    utswmed Repository record for Elucidating the Impact of Endogenous Proteins on Lipid Nanoparticle Organ-Targeting Outcomes (opens in a new tab)

  11. Development of Aerosolizable mRNA Incorporated Chitosan Nanoparticles for Efficient Lung Delivery

    Recently, the potential of messenger RNA (mRNA) as a promising therapeutic approach is beingunwrapped in the pulmonary medicine stream more than anywhere else. The unique function of directing the cells in protein synthesis “in vivo” has raised the mRNA as an effective alternative for a non-viral, …

    creighton Repository record for Development of Aerosolizable mRNA Incorporated Chitosan Nanoparticles for Efficient Lung Delivery (opens in a new tab)

  12. Degradable polymeric nano-films and particles as delivery platforms for vaccines and immunotherapeutics

    … by acting as platforms that facilitate the delivery of molecules to appropriate tissue and cellular locations to achieve therapeutic outcomes. To this end, we have designed and characterized nano-films and particles employing a hydrolytically degradable polymer for the delivery of vaccine …

    mit Repository record for Degradable polymeric nano-films and particles as delivery platforms for vaccines and immunotherapeutics (opens in a new tab)

  13. Engineering therapeutic messenger RNA for triple-negative breast cancer

    … by the use of viral vectors. Messenger RNA (mRNA) technology has been cemented as a safe and effective therapeutic modality, as evidenced by its success in SARS CoV-2 vaccination. Its potential to revolutionise cancer therapy is now becoming increasingly evident. We hypothesised that mRNA

    cork Repository record for Engineering therapeutic messenger RNA for triple-negative breast cancer (opens in a new tab)

  14. Design, Off-Target Toxicity, and Intracellular Processing of Nucleotide-Modified Messenger RNAs for Human Therapies

    … to gene therapy, in which in vitro-transcribed mRNA (IVT mRNA) is introduced to cells for therapeutic applications. IVT mRNA is transfected into target cells, translated, and the fully-synthesised encoded protein exerts a therapeutic effect via specific mechanisms. IVT mRNAs have been applied to …

    cambridge Repository record for Design, Off-Target Toxicity, and Intracellular Processing of Nucleotide-Modified Messenger RNAs for Human Therapies (opens in a new tab)

  15. Functionalized polypeptide- and lipid-based nanoparticles - synthesis and applications

    Submission published under a 24 month embargo labeled 'Closed Access', the embargo will last until 2025-08-01

    uiuc Repository record for Functionalized polypeptide- and lipid-based nanoparticles - synthesis and applications (opens in a new tab)

  16. ENGINEERING LIPID AND HYBRID NANOVECTORS FOR TARGETED GENE DELIVERY TO MACROPHAGE SUBPOPULATIONS

    In this work, a structural taxonomy of these systems is proposed with the aim of clarifying the possible architectures that can arise when genetic materials are complexed with a polycation or a lipid, followed by the addition of a lipid or a polymer to ameliorate the cellular toxicity, modulate …

    milano Repository record for ENGINEERING LIPID AND HYBRID NANOVECTORS FOR TARGETED GENE DELIVERY TO MACROPHAGE SUBPOPULATIONS (opens in a new tab)