Peptide-based gene delivery vectors

2019 ◽  
Vol 7 (11) ◽  
pp. 1824-1841 ◽  
Author(s):  
Ziyao Kang ◽  
Qingbin Meng ◽  
Keliang Liu
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Nucleic Acids ◽  
Delivery Systems ◽  
Disease Treatment ◽  
P Gene ◽  
Gene Delivery Vectors ◽  
Efficient Gene

Gene therapy as a strategy for disease treatment requires safe and efficient gene delivery systems that encapsulate nucleic acids and deliver them to effective sites in the cell.

Multivalent polyrotaxane vectors as adaptive cargo complexes for gene therapy

2018 ◽  
Vol 9 (7) ◽  
pp. 845-859 ◽  
Author(s):  
Rodinel Ardeleanu ◽  
Andrei I. Dascalu ◽  
Andrei Neamtu ◽  
Dragos Peptanariu ◽  
Cristina M. Uritu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery Systems ◽  
Efficient Gene

The philosophy to design and construct polyrotaxane carriers, as efficient gene delivery systems.

Effects of heterocyclic-based head group modifications on the structure–activity relationship of tocopherol-based lipids for non-viral gene delivery

2016 ◽  
Vol 14 (28) ◽  
pp. 6857-6870 ◽  
Author(s):  
Mallikarjun Gosangi ◽  
Thasneem Yoosuf Mujahid ◽  
Vijaya Gopal ◽  
Srilakshmi V. Patri
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Nucleic Acids ◽  
Viral Gene ◽  
Head Group ◽  
P Gene ◽  
Promising Strategy ◽  
Structure Activity ◽  
Viral Gene Delivery ◽  
Relationship Of

Gene therapy, a promising strategy for the delivery of therapeutic nucleic acids, is greatly dependent on the development of efficient vectors.

scholarly journals Non-Viral Gene Delivery Systems

Pharmaceutics ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 446
Author(s):  
Henrique Faneca
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Nucleic Acids ◽  
Delivery Systems ◽  
Human Diseases ◽  
Viral Gene ◽  
Viral Gene Delivery

The advances in the field of gene therapy have significantly improved the possibility for nucleic acids as highly promising agents for the treatment of both inherited and acquired human diseases [...]

scholarly journals Mesenchymal Stem Cells as a Gene Delivery Tool: Promise, Problems, and Prospects

Pharmaceutics ◽  
2021 ◽  
Vol 13 (6) ◽  
pp. 843
Author(s):  
Noha Attia ◽  
Mohamed Mashal ◽  
Gustavo Puras ◽  
Jose Luis Pedraz
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Gene Delivery ◽  
Nucleic Acids ◽  
Therapeutic Potential ◽  
Efficient Gene ◽  
Pros And Cons ◽  
Promise Problems ◽  
Viral Transfer

The cell-based approach in gene therapy arises as a promising strategy to provide safe, targeted, and efficient gene delivery. Owing to their unique features, as homing and tumor-tropism, mesenchymal stem cells (MSCs) have recently been introduced as an encouraging vehicle in gene therapy. Nevertheless, non-viral transfer of nucleic acids into MSCs remains limited due to various factors related to the main stakeholders of the process (e.g., nucleic acids, carriers, or cells). In this review, we have summarized the main types of nucleic acids used to transfect MSCs, the pros and cons, and applications of each. Then, we have emphasized on the most efficient lipid-based carriers for nucleic acids to MSCs, their main features, and some of their applications. While a myriad of studies have demonstrated the therapeutic potential for engineered MSCs therapy in various illnesses, optimization for clinical use is an ongoing challenge. On the way of improvement, genetically modified MSCs have been combined with various novel techniques and tools (e.g., exosomes, spheroids, 3D-Bioprinting, etc.,) aiming for more efficient and safe applications in biomedicine.

Exosome as a Natural Gene Delivery Vector for Cancer Treatment

2020 ◽  
Vol 20 (11) ◽  
pp. 821-830
Author(s):  
Prasad Pofali ◽  
Adrita Mondal ◽  
Vaishali Londhe
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Nucleic Acids ◽  
Cancer Treatment ◽  
Intestinal Barrier ◽  
Gene Carrier ◽  
Gene Delivery Vector ◽  
Delivery Vector

Background: Current gene therapy vectors such as viral, non-viral, and bacterial vectors, which are used for cancer treatment, but there are certain safety concerns and stability issues of these conventional vectors. Exosomes are the vesicles of size 40-100 nm secreted from multivesicular bodies into the extracellular environment by most of the cell types in-vivo and in-vitro. As a natural nanocarrier, exosomes are immunologically inert, biocompatible, and can cross biological barriers like the blood-brain barrier, intestinal barrier, and placental barrier. Objective: This review focusses on the role of exosome as a carrier to efficiently deliver a gene for cancer treatment and diagnosis. The methods for loading of nucleic acids onto the exosomes, advantages of exosomes as a smart intercellular shuttle for gene delivery and therapeutic applications as a gene delivery vector for siRNA, miRNA and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and also the limitations of exosomes as a gene carrier are all reviewed in this article. Methods: Mostly, electroporation and chemical transfection are used to prepare gene loaded exosomes. Results: Exosome-mediated delivery is highly promising and advantageous in comparison to the current delivery methods for systemic gene therapy. Targeted exosomes, loaded with therapeutic nucleic acids, can efficiently promote the reduction of tumor proliferation without any adverse effects. Conclusion: In the near future, exosomes can become an efficient gene carrier for delivery and a biomarker for the diagnosis and treatment of cancer.

scholarly journals Сationic liposomes as delivery systems for nucleic acids

2020 ◽  
Vol 15 (1) ◽  
pp. 7-27
Author(s):  
A. A. Mikheev ◽  
E. V. Shmendel ◽  
E. S. Zhestovskaya ◽  
G. V. Nazarov ◽  
M. A. Maslov
Keyword(s):  
Gene Therapy ◽  
Nucleic Acids ◽  
Serum Proteins ◽  
Transfection Efficiency ◽  
Biological Fluids ◽  
Genetic Material ◽  
Cationic Liposomes ◽  
Delivery Systems ◽  
Cationic Lipids

Objectives. Gene therapy is based on the introduction of genetic material into cells, tissues, or organs for the treatment of hereditary or acquired diseases. A key factor in the success of gene therapy is the development of delivery systems that can efficiently transfer genetic material to the place of their therapeutic action without causing any associated side effects. Over the past 10 years, significant effort has been directed toward creating more efficient and biocompatible vectors capable of transferring nucleic acids (NAs) into cells without inducing an immune response. Cationic liposomes are among the most versatile tools for delivering NAs into cells; however, the use of liposomes for gene therapy is limited by their low specificity. This is due to the presence of various biological barriers to the complex of liposomes with NA, including instability in biological fluids, interaction with serum proteins, plasma and nuclear membranes, and endosomal degradation. This review summarizes the results of research in recent years on the development of cationic liposomes that are effective in vitro and in vivo. Particular attention is paid to the individual structural elements of cationic liposomes that determine the transfection efficiency and cytotoxicity. The purpose of this review was to provide a theoretical justification of the most promising choice of cationic liposomes for the delivery of NAs into eukaryotic cells and study the effect of the composition of cationic lipids (CLs) on the transfection efficiency in vitro.Results. As a result of the analysis of the related literature, it can be argued that one of the most promising delivery systems of NAs is CL based on cholesterol and spermine with the addition of a helper lipid DOPE. In addition, it was found that varying the composition of cationic liposomes, the ratio of CL to NA, or the size and zeta potential of liposomes has a significant effect on the transfection efficiency.Conclusions. Further studies in this direction should include optimization of the conditions for obtaining cationic liposomes, taking into account the physicochemical properties and established laws. It is necessary to identify mechanisms that increase the efficiency of NA delivery in vitro by searching for optimal structures of cationic liposomes, determining the ratio of lipoplex components, and studying the delivery efficiency and properties of multicomponent liposomes.

Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler
Keyword(s):  
Gene Expression ◽  
Gene Therapy ◽  
Gene Transfer ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Adeno Associated Virus ◽  
Viral Genes ◽  
Virus Recombinant

AbstractAdeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression

Rational design of cationic cyclooligosaccharides as efficient gene delivery systems

2008 ◽  
pp. 2001 ◽  
Author(s):  
Alejandro Díaz-Moscoso ◽  
Patricia Balbuena ◽  
Marta Gómez-García ◽  
Carmen Ortiz Mellet ◽  
Juan M. Benito ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Rational Design ◽  
Delivery Systems ◽  
Efficient Gene

A dendritic catiomer with an MOF motif for the construction of safe and efficient gene delivery systems

2017 ◽  
Vol 5 (42) ◽  
pp. 8322-8329 ◽  
Author(s):  
Shuqi Dong ◽  
Qixian Chen ◽  
Wei Li ◽  
Zhu Jiang ◽  
Jianbiao Ma ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Gold Standard ◽  
Transfection Efficiency ◽  
A549 Cells ◽  
Delivery Systems ◽  
The Core ◽  
Efficient Gene

The dendritic catiomer using biocompatible Zr-MOFs as the core exhibited a markedly higher transfection efficiency and lower cytotoxicity than the commercial gold standard branched PEI25k in A549 cells.

scholarly journals A Physicochemical Approach for Predicting the Effectiveness of Peptide-Based Gene Delivery Systems for Use in Plasmid-Based Gene Therapy

1998 ◽  
Vol 74 (6) ◽  
pp. 2802-2814 ◽  
Author(s):  
John G. Duguid ◽  
Cynthia Li ◽  
Mei Shi ◽  
Mark J. Logan ◽  
Hector Alila ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery Systems ◽  
Physicochemical Approach
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