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  1. Intravitreal Delivery of PEGylated-ECO Plasmid DNA Nanoparticles for Gene Therapy of Stargardt Disease

    Objective

    Current gene therapy of inherited retinal diseases is achieved mainly by subretinal injection, which is invasive with severe adverse...

    Da Sun, Wenyu Sun, ... Zheng-Rong Lu in Pharmaceutical Research
    Article 05 March 2024
  2. Realizing time-staggered expression of nucleic acid-encoded proteins by co-delivery of messenger RNA and plasmid DNA on a single nanocarrier

    Co-delivery of different protein-encoding polynucleotide species with varying expression kinetics of their therapeutic product will become a...

    Sarah S. Nasr, Pascal Paul, ... Claus-Michael Lehr in Drug Delivery and Translational Research
    Article 15 July 2024
  3. Transfection with Plasmid-Encoding lncRNA-SLERCC nanoparticle-mediated delivery suppressed tumor progression in renal cell carcinoma

    Background

    The accumulating evidence confirms that long non-coding RNAs (lncRNAs) play a critical regulatory role in the progression of renal cell...

    Weipu Mao, Keyi Wang, ... Wei Li in Journal of Experimental & Clinical Cancer Research
    Article Open access 19 August 2022
  4. Modification of the tumor microenvironment enhances immunity with plasmid gene therapy

    Local intratumor delivery with electroporation of low levels of plasmids encoding molecules, induces an antitumor effect without causing systemic...

    Guilan Shi, Jody Synowiec, ... Richard Heller in Cancer Gene Therapy
    Article 09 February 2024
  5. Microfluidic Approaches for Gene Delivery and Therapy

    Gene editing techniques such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9) have been developed to target a gene into...
    Mayur Parekh, Zulfiqur Ali in Microfluidics in Pharmaceutical Sciences
    Chapter 2024
  6. Aerosolizable Plasmid DNA Dry Powders Engineered by Thin-film Freezing

    Purpose

    This study was designed to test the feasibility of using thin-film freezing (TFF) to prepare aerosolizable dry powders of plasmid DNA (pDNA)...

    Haiyue Xu, Chaeho Moon, ... Zhengrong Cui in Pharmaceutical Research
    Article 26 January 2023
  7. Investigation of the potential therapeutic effect of cationic lipoplex mediated fibroblast growth factor-2 encoding plasmid DNA delivery on wound healing

    Background

    Develo** an alternative and efficient therapy for wound healing has been an important research topic for pharmaceutical sciences. A...

    Gülşah Erel-Akbaba, Hasan Akbaba in DARU Journal of Pharmaceutical Sciences
    Article 07 September 2021
  8. ZIF-8 Nanoparticle: A Valuable Tool for Improving Gene Delivery in Sperm-Mediated Gene Transfer

    Metal–organic frameworks (MOFs) are porous materials with unique characteristics that make them well-suited for drug delivery and gene therapy...

    Marzieh Sameni, Parisa Moradbeigi, ... Mohammad Salehi in Biological Procedures Online
    Article Open access 26 January 2024
  9. Connexin-Containing Vesicles for Drug Delivery

    Connexin is a transmembrane protein present on the cell membrane of most cell types. Connexins assemble into a hexameric hemichannel known as...

    Mahmoud S. Hanafy, Zhengrong Cui in The AAPS Journal
    Article 24 January 2024
  10. Gene Delivery to Chondrocytes

    Delivering genes to chondrocytes offers new possibilities both clinically, for treating conditions that affect cartilage, and in the...
    Christopher V. Nagelli, Christopher H. Evans, Rodolfo E. De la Vega in Electromechanobiology of Cartilage and Osteoarthritis
    Chapter Open access 2023
  11. Nano–Bio Interactions: Exploring the Biological Behavior and the Fate of Lipid-Based Gene Delivery Systems

    Gene therapy for many diseases is rapidly becoming a reality, as demonstrated by the recent approval of various nucleic acid-based therapeutics....

    Seigo Kimura, Hideyoshi Harashima in BioDrugs
    Article 12 February 2024
  12. Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression

    Transcranial ultrasound combined with intravenous microbubbles can be used to increase blood-brain barrier permeability or, at lower pressures, to...

    Rikke Hahn Kofoed, Elizabeth M. Simpson, ... Isabelle Aubert in Gene Therapy
    Article 13 February 2023
  13. Viruses for Systemic Delivery

    Adeno-associated virus (AAV) is a nonpathogenic virus that has been leveraged as a gene-delivery vector due to its high transduction efficiency in...
    Zikai Wang, Alon Greenbaum, Jennifer B. Treweek in Vectorology for Optogenetics and Chemogenetics
    Protocol 2023
  14. Structure and Function of Cationic and Ionizable Lipids for Nucleic Acid Delivery

    Hereditary genetic diseases, cancer, and infectious diseases are affecting global health and become major health issues, but the treatment...

    Da Sun, Zheng-Rong Lu in Pharmaceutical Research
    Article 04 January 2023
  15. Polymeric Systems for Bioinspired Delivery of Angiogenic Molecules

    Growth factors are increasingly utilized to promote regeneration of lost or compromised tissues and organs. However, current strategies applying...
    Claudia Fischbach, David J. Mooney in Polymers for Regenerative Medicine
    Chapter
  16. The 60-year evolution of lipid nanoparticles for nucleic acid delivery

    Delivery of genetic information to the interior of target cells in vivo has been a major challenge facing gene therapies. This barrier is now being...

    P. R. Cullis, P. L. Felgner in Nature Reviews Drug Discovery
    Article 04 July 2024
  17. Controlled Delivery of Plasmid DNA to Melanoma Tumors by Gene Electrotransfer

    Gene electrotransfer (GET) is a reliable and effective physical method for in vivo delivery of plasmid DNA (pDNA). Several preclinical and clinical...
    Richard Heller, Guilan Shi in Melanoma
    Protocol 2021
  18. Delivery of nucleic acids using nanomaterials

    The increasing number of approved nucleic acid therapeutics demonstrates the potential for the prevention and treatment of a broad spectrum of...

    Yuyang Qin, Liyuan Ou, ... Ling Li in Molecular Biomedicine
    Article Open access 14 December 2023
  19. Efficient and scalable gene delivery method with easily generated cationic carbon dots

    Gene delivery is a complex process with several challenges when attempting to incorporate genetic material efficiently and safely into target cells....

    Manuel Algarra, Elena Gonzalez-Muñoz in Biological Procedures Online
    Article Open access 08 March 2024
  20. Pulmonary Delivery of Nucleic Acids

    Pulmonary diseases affect millions of people worldwide and elicit symptoms that may evolve into severe conditions. Despite currently recommended...
    Gemma Conte, Ivana d’Angelo, ... Francesca Ungaro in Respiratory Delivery of Biologics, Nucleic Acids, and Vaccines
    Chapter 2023
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