Gene Therapy and Gene Delivery Systems as Future Human Therapeutics

2005 ◽  
pp. 1-5 ◽  
Author(s):  
David V. Schaffer ◽  
Weichang Zhou
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery Systems

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

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

scholarly journals Gene delivery systems—gene therapy vectors for cystic fibrosis

2004 ◽  
Vol 3 ◽  
pp. 203-212 ◽  
Author(s):  
Daniel Klink ◽  
Dirk Schindelhauer ◽  
Andreas Laner ◽  
Torry Tucker ◽  
Zsuzsanna Bebok ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery Systems ◽  
Gene Therapy Vectors

scholarly journals Next Generation Delivery System for Proteins and Genes of Therapeutic Purpose: Why and How?

2014 ◽  
Vol 2014 ◽  
pp. 1-11 ◽  
Author(s):  
Ashish Ranjan Sharma ◽  
Shyamal Kumar Kundu ◽  
Ju-Suk Nam ◽  
Garima Sharma ◽  
C. George Priya Doss ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Delivery System ◽  
Delivery Systems ◽  
Therapeutic Protein ◽  
Gene Delivery System ◽  
Next Generation ◽  
Therapeutic Purpose ◽  
Detailed Explanation ◽  
Or Gene

Proteins and genes of therapeutic interests in conjunction with different delivery systems are growing towards new heights. “Next generation delivery systems” may provide more efficient platform for delivery of proteins and genes. In the present review, snapshots about the benefits of proteins or gene therapy, general procedures for therapeutic protein or gene delivery system, and different next generation delivery system such as liposome, PEGylation, HESylation, and nanoparticle based delivery have been depicted with their detailed explanation.

Pancreatic cancer: gene therapy approaches and gene delivery systems

2009 ◽  
Vol 10 (1) ◽  
pp. 73-88 ◽  
Author(s):  
Jin Xu ◽  
Chen Jin ◽  
Sijie Hao ◽  
Guopei Luo ◽  
Deliang Fu
Keyword(s):  
Pancreatic Cancer ◽  
Gene Therapy ◽  
Gene Delivery ◽  
Cancer Gene Therapy ◽  
Delivery Systems ◽  
Cancer Gene

scholarly journals Recent Advances in the Development of Bio-Reducible Polymers for Efficient Cancer Gene Delivery Systems

2019 ◽  
Vol 2 (1) ◽  
pp. 6-13 ◽  
Author(s):  
Kiel Sung Yong ◽  
◽  
Wan Kim Sung ◽  
◽  
◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Ribonucleic Acid ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Host Cells ◽  
Viral Gene ◽  
Cancer Gene ◽  
Inherited Disorders ◽  
Messenger Ribonucleic Acid

Gene therapy is the unique method for the use of genetic materials such as Messenger ribonucleic acid (mRNA), plasmid deoxyribonucleic acid (pDNA), and small interfering ribonucleic acid (siRNA) into specific host-cells for the treatment of inherited disorders in any diseases. The successful way to utilize the gene therapy is to develop the efficient cancer gene delivery systems. In this paper, the successful and efficient gene delivery systems are briefly reviewed on the basis of bio-reducible polymeric systems for cancer therapy. The viral gene delivery systems such as RNA-based viral and DNA-based viral vectors are also discussed. The development of bio-reducible polymer for gene delivery system has briefly discussed for the efficient cancer gene delivery of viral vectors and non-viral vectors.

scholarly journals Polymeric Nanoparticles in Gene Therapy: New Avenues of Design and Optimization for Delivery Applications

Polymers ◽  
2019 ◽  
Vol 11 (4) ◽  
pp. 745 ◽  
Author(s):  
Raj Rai ◽  
Saniya Alwani ◽  
Ildiko Badea
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Targeted Delivery ◽  
Polymeric Nanoparticles ◽  
Genetic Material ◽  
Delivery Systems ◽  
Viral Gene ◽  
Cationic Polymers ◽  
Natural Polymers ◽  
Viral Gene Delivery

The field of polymeric nanoparticles is quickly expanding and playing a pivotal role in a wide spectrum of areas ranging from electronics, photonics, conducting materials, and sensors to medicine, pollution control, and environmental technology. Among the applications of polymers in medicine, gene therapy has emerged as one of the most advanced, with the capability to tackle disorders from the modern era. However, there are several barriers associated with the delivery of genes in the living system that need to be mitigated by polymer engineering. One of the most crucial challenges is the effectiveness of the delivery vehicle or vector. In last few decades, non-viral delivery systems have gained attention because of their low toxicity, potential for targeted delivery, long-term stability, lack of immunogenicity, and relatively low production cost. In 1987, Felgner et al. used the cationic lipid based non-viral gene delivery system for the very first time. This breakthrough opened the opportunity for other non-viral vectors, such as polymers. Cationic polymers have emerged as promising candidates for non-viral gene delivery systems because of their facile synthesis and flexible properties. These polymers can be conjugated with genetic material via electrostatic attraction at physiological pH, thereby facilitating gene delivery. Many factors influence the gene transfection efficiency of cationic polymers, including their structure, molecular weight, and surface charge. Outstanding representatives of polymers that have emerged over the last decade to be used in gene therapy are synthetic polymers such as poly(l-lysine), poly(l-ornithine), linear and branched polyethyleneimine, diethylaminoethyl-dextran, poly(amidoamine) dendrimers, and poly(dimethylaminoethyl methacrylate). Natural polymers, such as chitosan, dextran, gelatin, pullulan, and synthetic analogs, with sophisticated features like guanidinylated bio-reducible polymers were also explored. This review outlines the introduction of polymers in medicine, discusses the methods of polymer synthesis, addressing top down and bottom up techniques. Evaluation of functionalization strategies for therapeutic and formulation stability are also highlighted. The overview of the properties, challenges, and functionalization approaches and, finally, the applications of the polymeric delivery systems in gene therapy marks this review as a unique one-stop summary of developments in this field.

Gene Therapy: Recent Advances and Applications in Dermatology

1996 ◽  
Vol 1 (2) ◽  
pp. 109-118 ◽  
Author(s):  
Michael J. Hope
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Skin Diseases ◽  
Ex Vivo ◽  
Delivery Systems ◽  
Nonviral Gene Delivery ◽  
Regulatory Sequences ◽  
Advantages And Disadvantages ◽  
Technical Advances ◽  
Near Future

Background: Gene therapy is an innovative and exciting new branch of medicine. Despite the fact that a human disease has yet to be cured using this therapeutic approach, numerous clinical trials are taking place around the world based on encouraging preclinical data. Objective: The aim of this review is to bring the reader up to date with this rapidly advancing field and to highlight the technical advances that must occur before gene therapy will become common practice in dermatology. Methods: The current level of gene delivery technology restricts the applications. The advantages and disadvantages of viral and nonviral gene delivery systems are discussed. Results: Considerable advances are being made in the areas of cancer immunotherapy and vaccines. Of particular importance to the treatment of skin diseases will be the isolation and ex vivo manipulation of epidermal stem cells, the development of skin-specific regulatory sequences for gene expression, and the formulation of gene delivery systems suitable for systemic administration. Conclusions: In general, skin and keratinocytes are considered to be good targets for gene transfer applications, and several diseases have been identified as potential candidates for treatment in the near future.

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