March Plasmid DNA-based gene therapy in neurological disorders. Munehisa Shimamura,; Hironori Nakagami,; Hitomi Kurinami,; Naoyuki Sato. The application of gene therapy in autoimmune disease represents a novel use of this technology. The goal of gene therapy in the treatment of. The aim of our book is to provide a detailed discussion of gene therapy application in human diseases. The book brings together major.


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This manuscript reviews the general methods by which genes are transferred as well as diverse examples of clinical applications acquired tissue damage, upper gastrointestinal tract infection, autoimmune disease, systemic protein deficiency.

Despite some well-publicized problems, gene therapy applications therapy has made substantive progress, including tangible success, albeit much slower than was initially predicted.

Gene Therapy Applications

Although gene therapy is still at a fairly primitive stage, it is firmly science based. Applications in Dentistry Bone repair Bone loss caused by trauma, neoplasia, reconstructive surgery, congenital defects or periodontal disease gene therapy applications a major worldwide health problem.


Regeneration of these bone structures would be enormously useful in gene therapy applications treatment of craniofacial and other bone anomalies, tooth loss, temporomandibular gene therapy applications other joint diseases, traumatic amputations and the consequences of tumor resection [ 1516 ].

The bone morphogenic proteins BMPs enable skeletal tissue formation during embryogenesis, growth, adulthood, and healing. Probably BMPs BMPs 2, 4 and 7 are the only growth factors which can singly induce de novo bone formation both in vitro and at heterotopic sites.


It will be possible to directly deliver the BMP-2 gene gene therapy applications vivo to tissues via an adeno viral vector to heal bone defects [ 25 ].

In one study genetically engineered mesenchymal stem cells expressing BMP-2 induced increased gene therapy applications of new blood vessels as well as new bone [ 6 ].

Michigan research group has found non osteogenic fibroblasts gingiva, dental pulp expresses BMP-7 gene after being infected with an adeno viral vector.

BMPs are agents well established in induction of both orthotopic and ectopic bone formation. In ex vivo studies, researchers accomplish the actual gene transfer in a tissue culture environment and then place the transduced cells carrying the foreign genes back into the host [ 6 ].

Transferring platelet-derived growth factor gene to periodontal cells results in DNA synthesis and cellular proliferation.

Delivery of PDGF by gene transfer has been shown to stimulate gingival fibroblast, PDL and tooth-lining cell cementoblast mitogenesis and gene therapy applications above that of gene therapy applications PDGF administration in vitro.

PDGF has also demonstrated positive effects in regenerating bone around teeth and dental implants [ 18 ].

Gene therapy: some history, applications, problems, and prospects.

This will facilitate localized regeneration of bone for periodontal and oral surgical procedures. Bone sialoprotein BSP is a major non collagenous protein in bone and other mineralized tissues.

By gene therapy applications in vivo delivery of a BSP gene into an osseous defect, it has been gene therapy applications to regenerate periodontal alveolar bone [ 4 ]. Studies have demonstrated the potential use of gene delivery to regenerate alveolar bone and cementum around teeth and alveolar bone associated with dental implant fixtures [ 18 ].

Ex vivo Gene Transfer for Bone Repair The advantage of an ex vivo gene transfer approach is that specific cells like bone marrow cells or stem cells can be selected as the cellular delivery vehicle for specific clinical problems.

Gene Therapy: Principles and Applications in Dentistry. | Open Access Journals

In addition, ex vivo strategies have a high efficiency of cell transduction. It is gene therapy applications to harvest cells from the patient, have a very short period of infection and reimplant the transduced cells at the appropriate anatomic site.

The cells that have received the most interest as a cellular delivery vehicle are mesenchymal stem cells, muscle-derived stem cells, adipose-derived stem cells, buffy coat cells from bone marrow or blood and skin fibroblasts [ 46 ].

Delivery of multiple genes gene therapy applications enhance bone repair.

  • Gene Therapy: Principles and Applications in Dentistry.

Tissue engineering Gene constructs, such as plasmid DNA or a viral particle are physically gene therapy applications within a matrix. Pain Managing or eliminating pain is a major part of dental practice.

The use of gene transfer technology offers a potentially novel approach to manipulate specific, localized biochemical pathways involved in pain generation. Gene transfer may be particularly useful for managing chronic and intractable pain.

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