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Robert Langer - One of the best experts on this subject based on the ideXlab platform.

  • Tissue-specific Gene Delivery via nanoparticle coating.
    Biomaterials, 2009
    Co-Authors: Todd J. Harris, Jordan J. Green, Peter W. Fung, Robert Langer, Daniel G. Anderson, Sangeeta N. Bhatia
    Abstract:

    The use of biomaterials for Gene Delivery can potentially avoid many of the safety concerns with viral Gene Delivery. However, the efficacy of polymeric Gene Delivery methods is low, particularly in vivo. One significant concern is that the interior and exterior composition of polymeric Gene Delivery nanoparticles are often coupled, with a single polymer backbone governing all functions from biophysical properties of the polymer/DNA particle to DNA condensation and release. In this work we develop electrostatically adsorbed poly(glutamic acid)-based peptide coatings to alter the exterior composition of a core Gene Delivery particle and thereby affect tissue-specificity of Gene Delivery function in vivo. We find that with all coating formulations tested, the coatings reduce potential toxicity associated with uncoated cationic Gene Delivery nanoparticles following systemic injection. Particles coated with a low 2.5:1 peptide:DNA weight ratio (w/w) form large 2 micron sized particles in the presence of serum that can facilitate specific Gene Delivery to the liver. The same particles coated at a higher 20:1 w/w form small 200 nm particles in the presence of serum that can facilitate specific Gene Delivery to the spleen and bone marrow. Thus, variations in nanoparticle peptide coating density can alter the tissue-specificity of Gene Delivery in vivo.

  • design of imidazole containing endosomolytic biopolymers for Gene Delivery
    Biotechnology and Bioengineering, 2000
    Co-Authors: Daniel W Pack, David Putnam, Robert Langer
    Abstract:

    The development of safe and effective Gene Delivery agents poses a great challenge in the quest to make human Gene therapy a reality. Cationic polymers represent one important class of materials for Gene Delivery, but to date they have shown only moderate efficiency. Improving the efficiency will require the design of new polymers incorporating optimized Gene Delivery properties. For example, inefficient release of the DNA/polymer complex from endocytic vesicles into the cytoplasm is one of the primary causes of poor Gene Delivery. Here we report the synthesis of a biocompatible, imidazole-containing polymer designed to overcome this obstacle. DNA/polymer polyplexes incorporating this polymer were shown to have desirable physico-chemical properties for Gene Delivery and are essentially nontoxic. Using this system, mammalian cells in vitro were transfected in the absence of any exogenous endosomolytic agent such as chloroquine. © 2000 John Wiley & Sons, Inc. Biotechnol Bioeng 67: 217–223, 2000.

  • Design of imidazole‐containing endosomolytic biopolymers for Gene Delivery
    Biotechnology and bioengineering, 2000
    Co-Authors: Daniel W Pack, David Putnam, Robert Langer
    Abstract:

    The development of safe and effective Gene Delivery agents poses a great challenge in the quest to make human Gene therapy a reality. Cationic polymers represent one important class of materials for Gene Delivery, but to date they have shown only moderate efficiency. Improving the efficiency will require the design of new polymers incorporating optimized Gene Delivery properties. For example, inefficient release of the DNA/polymer complex from endocytic vesicles into the cytoplasm is one of the primary causes of poor Gene Delivery. Here we report the synthesis of a biocompatible, imidazole-containing polymer designed to overcome this obstacle. DNA/polymer polyplexes incorporating this polymer were shown to have desirable physico-chemical properties for Gene Delivery and are essentially nontoxic. Using this system, mammalian cells in vitro were transfected in the absence of any exogenous endosomolytic agent such as chloroquine. © 2000 John Wiley & Sons, Inc. Biotechnol Bioeng 67: 217–223, 2000.

Daniel W Pack - One of the best experts on this subject based on the ideXlab platform.

  • design and development of polymers for Gene Delivery
    Nature Reviews Drug Discovery, 2005
    Co-Authors: Daniel W Pack, Allan S Hoffman, Patrick S Stayton
    Abstract:

    The lack of safe and efficient Gene-Delivery methods is a limiting obstacle to human Gene therapy. Synthetic Gene-Delivery agents, although safer than recombinant viruses, Generally do not possess the required efficacy. In recent years, a variety of effective polymers have been designed specifically for Gene Delivery, and much has been learned about their structure–function relationships. With the growing understanding of polymer Gene-Delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based Gene-Delivery systems will become an important tool for human Gene therapy.

  • design of imidazole containing endosomolytic biopolymers for Gene Delivery
    Biotechnology and Bioengineering, 2000
    Co-Authors: Daniel W Pack, David Putnam, Robert Langer
    Abstract:

    The development of safe and effective Gene Delivery agents poses a great challenge in the quest to make human Gene therapy a reality. Cationic polymers represent one important class of materials for Gene Delivery, but to date they have shown only moderate efficiency. Improving the efficiency will require the design of new polymers incorporating optimized Gene Delivery properties. For example, inefficient release of the DNA/polymer complex from endocytic vesicles into the cytoplasm is one of the primary causes of poor Gene Delivery. Here we report the synthesis of a biocompatible, imidazole-containing polymer designed to overcome this obstacle. DNA/polymer polyplexes incorporating this polymer were shown to have desirable physico-chemical properties for Gene Delivery and are essentially nontoxic. Using this system, mammalian cells in vitro were transfected in the absence of any exogenous endosomolytic agent such as chloroquine. © 2000 John Wiley & Sons, Inc. Biotechnol Bioeng 67: 217–223, 2000.

  • Design of imidazole‐containing endosomolytic biopolymers for Gene Delivery
    Biotechnology and bioengineering, 2000
    Co-Authors: Daniel W Pack, David Putnam, Robert Langer
    Abstract:

    The development of safe and effective Gene Delivery agents poses a great challenge in the quest to make human Gene therapy a reality. Cationic polymers represent one important class of materials for Gene Delivery, but to date they have shown only moderate efficiency. Improving the efficiency will require the design of new polymers incorporating optimized Gene Delivery properties. For example, inefficient release of the DNA/polymer complex from endocytic vesicles into the cytoplasm is one of the primary causes of poor Gene Delivery. Here we report the synthesis of a biocompatible, imidazole-containing polymer designed to overcome this obstacle. DNA/polymer polyplexes incorporating this polymer were shown to have desirable physico-chemical properties for Gene Delivery and are essentially nontoxic. Using this system, mammalian cells in vitro were transfected in the absence of any exogenous endosomolytic agent such as chloroquine. © 2000 John Wiley & Sons, Inc. Biotechnol Bioeng 67: 217–223, 2000.

Alexander M Seifalian - One of the best experts on this subject based on the ideXlab platform.

  • Will Nanotechnology Bring New Hope for Gene Delivery?
    Trends in Biotechnology, 2017
    Co-Authors: Joanna K.l. Wong, Nagy Habib, Rashin Mohseni, Robert E Maclaren, Amir Ali Hamidieh, Alexander M Seifalian
    Abstract:

    The high mortality rates of cancer patients receiving standard treatments emphasize the crucial need for alternative treatments. One promising strategy is to use organic nanoparticles (NPs) for Gene Delivery. Although multifunctional NPs theoretically have many desirable properties as Gene vectors, there are several practical barriers to successful Gene Delivery. In this review we discuss the properties of NPs and overview in vitro and preclinical studies of organic NPs over the past 5 years. The results of recent clinical trials suggest that the NP field holds significant promise. The annual market value for successful Gene Delivery could exceed US$30 billion, and this will encourage researchers to study the application of NPs to therapeutic Gene Delivery.

Jeong H Park - One of the best experts on this subject based on the ideXlab platform.

  • Ultrasound-mediated Gene Delivery
    Expert opinion on drug delivery, 2010
    Co-Authors: Chang S Yoon, Jeong H Park
    Abstract:

    Importance of the field: The use of ultrasound with microbubbles raises the possibility of an efficient and safe Gene Delivery.Areas covered in this review: This review summarizes the current state of the art of Gene Delivery by sonoporation under the following topics. First, the basic ultrasound parameters and the characteristics of microbubble in biological systems are discussed. Second, the extensions of sonoporation to other fields of Gene Delivery such as viral and non-viral vector are briefly reviewed. Finally, recent applications in an animal model for various diseases are introduced.What the reader will gain: Information and comments on Gene Delivery by sonoporation or enhanced cell membrane permeability by means of ultrasound.Take home message: Ultrasound-mediated Gene Delivery combined with microbubble agents provides significant safety advantages over other methods of local Gene Delivery.

Yuehe Lin - One of the best experts on this subject based on the ideXlab platform.

  • Recent progress in nanomaterials for Gene Delivery applications
    Biomaterials science, 2016
    Co-Authors: Erhan Keles, Yang Song, Wen-ji Dong, Yuehe Lin
    Abstract:

    Nanotechnology-based Gene Delivery is the division of nanomedicine concerned with the synthesis, characterization, and functionalization of nanomaterials to be used in targeted-Gene Delivery applications. Nanomaterial-based Gene Delivery systems hold great promise for curing fatal inherited and acquired diseases, including neurological disorders, cancer, cardiovascular diseases, and acquired immunodeficiency syndrome (AIDS). However, their use in clinical applications is still controversial. To date, the Food and Drug Administration (FDA) has not approved any Gene Delivery system because of the unknown long-term toxicity and the low Gene transfection efficiency of nanomaterials in vivo. Compared to viral vectors, nonviral Gene Delivery vectors are characterized by a low preexisting immunogenicity, which is important for preventing a severe immune response. In addition, nonviral vectors provide higher loading capacity and ease of fabrication. For these reasons, this review article focuses on applications of nonviral Gene Delivery systems, including those based on lipids, polymers, graphene, and other inorganic nanoparticles, and discusses recent advances in nanomaterials for Gene therapy. Methods of synthesizing these nanomaterials are briefly described from a materials science perspective. Also, challenges, critical issues, and concerns about the in vivo applications of nanomaterial-based Gene Delivery systems are discussed. It should be noted that this article is not a comprehensive review of the literature.