Nowadays, gene delivery for therapeutic objects is considered one of the most promising strategies to cure both the genetic and acquired diseases of human. The design of efficient gene delivery vectors possessing the high transfection efficiencies and low cytotoxicity is considered the major challenge for delivering a target gene to specific tissues or cells. On this base, the investigations on...

for electronic indexing) The delivery of a therapeutic gene inserted in a vector for somatic gene therapy holds great promise for the future. Encouraging results from the clinical trials of first-generation plasmid-based medicines/ vaccines have further validated the importance of molecular biology and biotechnology research. With newer developments in bioengineering for the mass production and...

The engineering of novel properties and functions into viral vectors for improved gene delivery remains a barrier to the development of efficient, customized gene delivery vehicles. Rational methods for designing improved viral vectors are often experimentally challenging and laborious, particularly when knowledge of viral structure-function relationships is limited. As an alternative, high-thr...

The ability to mediate targeted and specific delivery of therapeutics to cancer cells remains one of the most important hurdles in effectively treating cancer. This aspect also remains as one of the greatest limitations of gene therapy as well. Targeted vectors based on the use of DNA-binding agents attached to cell specific ligands or "molecular conjugates" were created with the goal of over-c...

PURPOSE Non-viral vectors have been widely proposed as safer alternatives to viral vectors, and cationic polymers have gained increasing attention because they can form self-assembly with DNA. Chitosan is also considered to be a good candidate for gene delivery systems, since it is already known as a biocompatible, biodegradable, and low toxic material with high cationic potential. However, low...

Non-viral gene delivery holds great promise for promoting tissue regeneration, and offers a potentially safer alternative than viral vectors. Great progress has been made to develop biodegradable polymeric vectors for non-viral gene delivery in 2D culture, which generally involves isolating and modifying cells in vitro, followed by subsequent transplantation in vivo. Scaffold-mediated gene deli...

Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clotting. Gene therapy hemophilia involves introduction healthy gene into patient's cells to produce missing protein. There are two main types hemophilia: ex vivo therapy, which extraction from patient, these in laboratory, and reintroduction modified patient; direct delivery body using viral vector...

The cellular heterogeneity and complex circuitry of the central nervous system make it difficult to achieve precise delivery of experimental and therapeutic agents. We report here an in vivo retrograde gene delivery strategy to target mature projection neurons using adeno-associated virus, a vector with low toxicity and the capacity for long-term gene expression. Viral delivery to axon terminal...

Currently, 33 gene-therapy drugs/products have been approved in the clinic. Over 3000 completed and ongoing clinical gene therapy trials reported worldwide. The development/maturation of tools for manipulation delivery, as well molecular advances diagnosis genetic diseases, played a central role therapy, which greatly revolutionized field. Versatile diverse manipulations deliveries, with possib...