Advanced Materials for Gene Delivery

2014 ◽  
Vol 995 ◽  
pp. 29-47 ◽  
Author(s):  
Mohammad A. Jafar Mazumder ◽  
Md. Hasan Zahir ◽  
Sharif F. Zaman
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Genetic Disorders ◽  
Basic Research ◽  
Advanced Materials ◽  
Clinical Settings ◽  
Efficient Gene ◽  
Efficient Delivery ◽  
Promising Treatment

Gene therapy is a widespread and promising treatment of many diseases resulting from genetic disorders, infections and cancer. The feasibility of the gene therapy is mainly depends on the development of appropriate method and suitable vectors. For an efficient gene delivery, it is very important to use a carrier that is easy to produce, stable, non-oncogenic and non-immunogenic. Currently most of the vectors actually suffer from many problems. Therefore, the ideal gene therapy delivery system should be developed that can be easily used for highly efficient delivery and able to maintain long-term gene expression, and can be applicable to basic research as well as clinical settings. This article provides a brief over view on the concept and aim of gene delivery, the different gene delivery systems and use of different materials as a carrier in the area of gene therapy.

scholarly journals Sterile Abscess in the Myocardium after Direct Intramyocardial Injection Related to Gene Therapy in a Swine Model

2011 ◽  
Vol 2011 ◽  
pp. 1-2 ◽  
Author(s):  
Kiyotake Ishikawa ◽  
Dennis Ladage ◽  
Lisa Tilemann ◽  
Yoshiaki Kawase ◽  
Roger J. Hajjar
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Viral Vectors ◽  
Swine Model ◽  
Clinical Settings ◽  
Intramyocardial Injection ◽  
New Therapy ◽  
Sterile Abscess ◽  
Efficient Gene ◽  
Cardiac Gene

Cardiac gene therapy is one of the most promising approaches to cure patients with cardiac dysfunctions. Many ways of efficient gene transfer using viral vectors are tested, and some of them are already used in clinical settings. However, it is always important to be keenly alert to the possible complications when a new therapy is introduced. We present a case of myocardial sterile abscess in a swine model associated with a direct myocardial injection.

scholarly journals Gene Therapy: The Molecular Bandage for Treating Genetic Disorders

1970 ◽  
Vol 3 (1) ◽  
pp. 24-27
Author(s):  
Md Manjurul Karim
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Genetic Disorders ◽  
Clinical Status ◽  
Genetic Material ◽  
Review Article ◽  
Cell Tissue ◽  
Efficient Gene ◽  
Effective Delivery ◽  
A Cell

The concept of gene therapy involves the transfer of genetic material into a cell, tissue, or whole organ, with a view to curing a disease or at least improving the clinical status of a patient. Much of its success relies heavily on the development of an effective delivery system that is capable of efficient gene transfer in a variety of tissues, without causing any associated pathogenic effects. Viral vectors currently offer the best choice for efficient gene delivery, what has been discussed in this review article. Their performance and pathogenecity has been evaluated in animal models, and encouraging results form the basis for clinical trials to treat genetic disorders and acquired diseases. Despite some initial success in these trials, vector development remains a seminal concern for improved gene therapy technologies. DOI: http://dx.doi.org/10.3329/akmmcj.v3i1.10110 AKMMCJ 2012; 3(1): 24-27

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 Genetic rescue models refute nonautonomous rod cell death in retinitis pigmentosa

2017 ◽  
Vol 114 (20) ◽  
pp. 5259-5264 ◽  
Author(s):  
Susanne F. Koch ◽  
Jimmy K. Duong ◽  
Chun-Wei Hsu ◽  
Yi-Ting Tsai ◽  
Chyuan-Sheng Lin ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Retinitis Pigmentosa ◽  
High Efficiency ◽  
Genetic Rescue ◽  
Rod Photoreceptors ◽  
Rods And Cones ◽  
Promising Treatment ◽  
Low Efficiency ◽  
Rod Cell

Retinitis pigmentosa (RP) is an inherited neurodegenerative disease, in which the death of mutant rod photoreceptors leads secondarily to the non-cell autonomous death of cone photoreceptors. Gene therapy is a promising treatment strategy. Unfortunately, current methods of gene delivery treat only a fraction of diseased cells, yielding retinas that are a mosaic of treated and untreated rods, as well as cones. In this study, we created two RP mouse models to test whether dying, untreated rods negatively impact treated, rescued rods. In one model, treated and untreated rods were segregated. In the second model, treated and untreated rods were diffusely intermixed, and their ratio was controlled to achieve low-, medium-, or high-efficiency rescue. Analysis of these mosaic retinas demonstrated that rescued rods (and cones) survive, even when they are greatly outnumbered by dying photoreceptors. On the other hand, the rescued photoreceptors did exhibit long-term defects in their outer segments (OSs), which were less severe when more photoreceptors were treated. In summary, our study suggests that even low-efficiency gene therapy may achieve stable survival of rescued photoreceptors in RP patients, albeit with OS dysgenesis.

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.

Tailoring highly branched poly(β-amino ester)s: a synthetic platform for epidermal gene therapy

2015 ◽  
Vol 51 (40) ◽  
pp. 8473-8476 ◽  
Author(s):  
Jian-Yuan Huang ◽  
Yongsheng Gao ◽  
Lara Cutlar ◽  
Jonathan O'Keeffe-Ahern ◽  
Tianyu Zhao ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Human Keratinocytes ◽  
Amino Ester ◽  
Efficient Gene

Highly branched poly(β-amino ester)s were synthesised for safer and more efficient gene delivery to human keratinocytes than commercial transfection reagents.

Peptide Shuttle-Mediated Delivery for Brain Gene Therapies

2020 ◽  
Vol 20 (32) ◽  
pp. 2945-2958
Author(s):  
Josep Garcia ◽  
Pol Arranz-Gibert ◽  
Macarena Sánchez-Navarro ◽  
Ernest Giralt ◽  
Meritxell Teixidó
Keyword(s):  
Gene Therapy ◽  
Special Focus ◽  
Gene Therapies ◽  
Efficient Gene ◽  
Wide Range ◽  
Monogenic Diseases ◽  
Delivery Strategies ◽  
Inpatient Safety ◽  
Broad Overview

The manipulation of an individual’s genetic information to treat a disease has revolutionized the biomedicine field. Despite the promise of gene therapy, this treatment can have long-term sideeffects. Efforts in the field and recent discoveries have already led to several improvements, including efficient gene delivery and transfer, as well as inpatient safety. Several studies to treat a wide range of pathologies-such as cancer or monogenic diseases- are currently being conducted. Here we provide a broad overview of methodologies available for gene therapy, placing a strong emphasis on treatments for central nervous system diseases. Finally, we give a perspective on current delivery strategies to treat such diseases, with a special focus on systems that use peptides as delivery vectors.

Ligand targeting and peptide functionalized polymers as non-viral carriers for gene therapy

2020 ◽  
Vol 8 (1) ◽  
pp. 64-83 ◽  
Author(s):  
Khan Muhammad ◽  
Jing Zhao ◽  
Ihsan Ullah ◽  
Jintang Guo ◽  
Xiang-kui Ren ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Functionalized Polymers ◽  
Gene Carriers ◽  
Efficient Gene

Ligand targeting and peptide functionalized polymers serve as gene carriers for efficient gene delivery.

scholarly journals Septic acute kidney injury: a review of basic research

2020 ◽  
Vol 24 (12) ◽  
pp. 1091-1102 ◽  
Author(s):  
Daisuke Nakano
Keyword(s):  
Acute Kidney Injury ◽  
Molecular Mechanisms ◽  
Functional Decline ◽  
Kidney Injury ◽  
Basic Research ◽  
Clinical Settings ◽  
Long Term Effects ◽  
Research Information ◽  
Septic Acute Kidney Injury

Abstract Sepsis is a major cause of acute kidney injury (AKI) among patients in the intensive care unit. However, the numbers of basic science papers for septic AKI account for only 1% of all publications on AKI. This may be partially attributable to the specific pathophysiology of septic AKI as compared to that of the other types of AKI because it shows only modest histological changes despite functional decline and often requires real-time functional analysis. To increase the scope of research in this field, this article reviews the basic research information that has been reported thus far on the subject of septic AKI, mainly from the viewpoint of functional dysregulation, including some knowledge acquired with multiphoton intravital imaging. Moreover, the efficacy and limitation of the potential novel therapies are discussed. Finally, the author proposes several points that should be considered when designing the study, such as monitoring the long-term effects of the intervention and reflecting the clinical settings for identifying the molecular mechanisms and for challenging the intervention effects.

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