Research Progress of nucleic acid delivery vectors for gene therapy

2020 ◽  
Vol 22 (1) ◽  
Author(s):  
Yang Jiao ◽  
Zhang Li Xia ◽  
Li Jiang Ze ◽  
Hui Jing ◽  
Bai Xin ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Research Progress ◽  
Nucleic Acid Delivery

scholarly journals Role of Lipid-Based and Polymer-Based Non-Viral Vectors in Nucleic Acid Delivery for Next-Generation Gene Therapy

Molecules ◽  
2020 ◽  
Vol 25 (12) ◽  
pp. 2866 ◽  
Author(s):  
Aniket Wahane ◽  
Akaash Waghmode ◽  
Alexander Kapphahn ◽  
Karishma Dhuri ◽  
Anisha Gupta ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Messenger Rna ◽  
Viral Vectors ◽  
Cationic Lipids ◽  
Nucleic Acid Delivery ◽  
Nucleic Acid Analogs ◽  
Organ Specific ◽  
Interfering Rna

The field of gene therapy has experienced an insurgence of attention for its widespread ability to regulate gene expression by targeting genomic DNA, messenger RNA, microRNA, and short-interfering RNA for treating malignant and non-malignant disorders. Numerous nucleic acid analogs have been developed to target coding or non-coding sequences of the human genome for gene regulation. However, broader clinical applications of nucleic acid analogs have been limited due to their poor cell or organ-specific delivery. To resolve these issues, non-viral vectors based on nanoparticles, liposomes, and polyplexes have been developed to date. This review is centered on non-viral vectors mainly comprising of cationic lipids and polymers for nucleic acid-based delivery for numerous gene therapy-based applications.

scholarly journals Effective lung-targeted RNAi in mice with peptide-based delivery of nucleic acid

2019 ◽  
Vol 9 (1) ◽  
Author(s):  
Kaido Kurrikoff ◽  
Krista Freimann ◽  
Kadi-Liis Veiman ◽  
Elin Madli Peets ◽  
Andres Piirsoo ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Lung Disease ◽  
Lung Tissue ◽  
Lung Inflammation ◽  
Disease Models ◽  
Nucleic Acid Delivery ◽  
Acute Lung Inflammation ◽  
Disease Symptoms ◽  
Targeted Gene Therapy

AbstractWe have previously developed efficient peptide-based nucleic acid delivery vectors PF14 and NF55, where we have shown that these vectors preferentially transfect lung tissue upon systemic administration with the nucleic acid. In the current work, we have explored the utilization and potential of these vectors for the lung-targeted gene therapy. Accordingly, we assessed the efficacy of these peptides in (i) two different lung disease models – acute lung inflammation and asthma in mice and (ii) using two different nucleic acid cargos – siRNA and pDNA encoding shRNA. Using RNAi against cytokine TNFα, we showed efficient anti-inflammatory effects in both disease models and observed decreased disease symptoms. Our results highlight the potential of our transfection vectors for lung gene therapy.

Complexation of short ds RNA/DNA oligonucleotides with Gemini micelles: a time resolved SAXS and computational study

2017 ◽  
Vol 19 (4) ◽  
pp. 3046-3055 ◽  
Author(s):  
Sara Falsini ◽  
Emanuela Di Cola ◽  
Martin In ◽  
Maria Giordani ◽  
Stefano Borocci ◽  
...  
Keyword(s):  
Gene Expression ◽  
Gene Therapy ◽  
Nucleic Acid ◽  
Computational Study ◽  
Nucleic Acid Delivery ◽  
Time Resolved ◽  
Dna Oligonucleotides ◽  
P Gene

Gene therapy is based on nucleic acid delivery to pathogenic cells in order to modulate their gene expression.

OPTIMIZATION OF THE TECHNOLOGY OF BINDING COMPONENT PREPARATION FOR CATIONIC LIPOSOMES IN GENE THERAPY

2020 ◽  
pp. 169-171
Author(s):  
D. Egorov ◽  
M. Maslov
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Cationic Liposomes ◽  
Mitsunobu Reaction ◽  
Synthetic Approach ◽  
Nucleic Acid Delivery ◽  
Novel Approach ◽  
Binding Component

Cationic liposomes for nucleic acid delivery were prepared from dimeric polycationic amphiphile. Novel synthetic approach was developed for that. Comparison of both novel approach based on Mitsunobu reaction and previous one is provided.

scholarly journals Methodologies to investigate intracellular barriers for nucleic acid delivery in non-viral gene therapy

Nano Today ◽  
2018 ◽  
Vol 21 ◽  
pp. 74-90 ◽  
Author(s):  
Lotte M.P. Vermeulen ◽  
Toon Brans ◽  
Stefaan C. De Smedt ◽  
Katrien Remaut ◽  
Kevin Braeckmans
Keyword(s):  
Gene Therapy ◽  
Nucleic Acid ◽  
Viral Gene ◽  
Nucleic Acid Delivery ◽  
Viral Gene Therapy

Polyethylenimine-mediated impairment of mitochondrial membrane potential, respiration and membrane integrity: Implications for nucleic acid delivery and gene therapy

Mitochondrion ◽  
2012 ◽  
Vol 12 (1) ◽  
pp. 162-168 ◽  
Author(s):  
Anna K. Larsen ◽  
Dominika Malinska ◽  
Izabela Koszela-Piotrowska ◽  
Ladan Parhamifar ◽  
A. Christy Hunter ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Membrane Potential ◽  
Nucleic Acid ◽  
Mitochondrial Membrane Potential ◽  
Mitochondrial Membrane ◽  
Membrane Integrity ◽  
Nucleic Acid Delivery

scholarly journals 1,28-Di[(cholest-5-en-3β-yl)disulfanyl]-4,25-dioxo-3,8,12,17,21,26-hexaazaoctacosane tetrahydrochloride

2018 ◽  
Author(s):  
Pavel A. Puchkov ◽  
Elena V. Shmendel ◽  
Valeria D. Andreeva ◽  
Nina G. Morozova ◽  
Marina A. Zenkova ◽  
...  
Keyword(s):  
Thin Film ◽  
Gene Therapy ◽  
Nucleic Acid ◽  
Transfection Efficiency ◽  
Cationic Liposomes ◽  
Hek293 Cells ◽  
Nucleic Acid Delivery ◽  
Promising Candidate ◽  
Effective Delivery

The absence of highly effective delivery systems is a major challenge for gene therapy. Our work was aimed at the development of novel cationic liposomes possessing high transfection efficiency. For this purpose, a novel disulfide polycationic amphiphile 2S4 was synthesized. Cationic liposomes based on 2S4 and a helper lipid DOPE were formed by the thin film hydration method and exhibited effective pDNA delivery into the HEK293 cells, with a maximal transfection activity superior to that of the commercial agent Lipofectamine® 2000. Our results suggest that the polycationic amphiphile 2S4 is a promising candidate for in vitro nucleic acid delivery.

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