Genes and Gene Therapy

Genes are the building blocks of inheritance. Passed from parent to child, they contain instructions for making proteins. If genes don't produce the right proteins or produce them correctly, a child can have a genetic disorder.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include

Swapping an abnormal gene for a normal one
Repairing an abnormal gene
Altering the degree to which a gene is turned on or off

Although there is much hope for gene therapy, it is still experimental.

Genetics Home Reference

Gene Therapy on Wikipedia

vector. A new gene is inserted into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein. '''Gene therapy''' is the insertion of genes into an individual's cell (biology) and Biological tissues to treat a disease, and hereditary diseases in particular. Gene therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is often lumped together with them.

Background

In the 1980s, advances in molecular biology had already enabled human genes to be sequencing and cloning. Scientists looking for a method of easily producing proteins, such as the protein deficient in diabetics — insulin, investigated introducing human genes to bacteria DNA. The modified bacteria then produce the corresponding protein, which can be harvested and injected in people who cannot produce it naturally. Scientists took the logical step of trying to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia. However, this has been much harder than modifying simple bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering it to the right site on the genome.

Types of gene therapy

In theory it is possible to transform either somatic cells (most cells of the body) or cells of the germline (such as stem cells, sperm and Ovums). All gene therapy so far in people has been directed at somatic cells, whereas germline engineering in humans ... [ Read More ]

External Resources

NjeX - Medical Device Implanting Technology - Implants for pharmaceutical and gene therapy --- controlled release pellets, brachytherapy seeds, and chip and nanodevices.

Aastrom Biosciences, Inc. - Develops proprietary process technologies and devices for cell therapy applications, including stem cell therapies and gene therapy.

Qiagen - Products and services for life science research, molecular diagnostics, genomics, and gene therapy.

Thorpe, P. H. - Studies of Cystic Fibrosis gene therapy. From the University of Edinburgh in Scotland.

Tronolab Website - Collection of protocols, maps and sequences regarding manipulation of lentiviral vectors, siRNA and transgenic animals. Applications in HIV, stem cell and gene therapy research at University of Geneva.

Genetic Vaccines and Therapy - Peer-reviewed, open access online articles on clinical and basic research in pathogen genes for vaccines and targets for therapy, novel transfer vectors and methods of delivery in treating human disease.

Laboratory of Transplantation Immunology - Confronting research in human immunology, gene therapy, non-viral vector peptide vaccines, cell adhesion and mucosal immunology for therapeutic application at the University of Nebraska Medical Center, Omaha, NE.

Molecular Microbiology and Immunology-SLU - Ph.D. degree training focusing on host-pathogen interactions, include virology, viral pathology, biodefense, cancer gene therapy, and vaccine development. Site includes information on curriculum and faculty research.

Peixuan Guo - Research on mechanism of viral assembly, DNA packaging, molecular vaccines, antiviral therapy, gene delivery, and chimerical virus displaying antigens or antibodies.

Special Project Angiogenesis - Offers a look at angiostatic therapy, cancer, genes, publications, and lab news. Includes goals, tools, and participants at the First Interdisciplinary Scientific Project on Tumor Angiogenesis sponsored by Italian Association for Cancer Research.

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