Bioinformatics
BIOINFORMATICS IN
GENE THERAPY
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What's gene therapy?
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Gene therapy is essentially a technique in which scientists utilize genes in order to treat or prevent disease. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. In order to do this, scientists commonly use viruses to carry the genetic information into cells. These viruses have been genetically altered to carry normal human DNA. Scientists can then use viruses to add or replace an abnormal gene to improve the body's ability to fight disease.
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gene therapy has been use to:
Replace Mutated Genes
Overtime, certain genes begin to work improperly or shut down and not work at all. With gene therapy, scientists can replace these defective genes to ensure that the mutated cells die and to make certain that they will never come back again.
Replace Mutated Genes
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Treat
Cancer
Cancer is basically an uncontrolled group of cells that grow in organs often resulting in organ failure and death. Scientists are currently working on methods for gene therapy to cure cancer.
Treat
Cancer
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Treat Immune Deficiencies
With gene therapy, many inherited immune deficiencies have been successfully treated. Retroviruses are often used in order to deliver the normal copies of the irregular genes. Ensuring that the virus will infect and transfer the gene to the targeted cell, the stem cells have been treated outside of the person's body.
Treat Immune Deficiencies
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Treat Parkinson's Disease
Parkinson's Disease is a long-term progressive disorder that affects the motor system of the central nervous system. In an experiment, a small group of people who had advanced Parkinson's Disease were introduced three genes into cells in a small area of the brain by retroviruses. After treatment, patients had improved muscle control.
Treat Parkinson's Disease
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Treat Hemophilia
Hemophilia is a rare genetic disorder that causes the inability to form blood clots. This is due to the lack of, or missing proteins that allow for blood to clot. Researchers successfully used a viral vector to deliver a gene for Factor IX, the missing clotting protein, to liver cells. After treatment, most of the patients made at least some Factor IX, and they had fewer bleeding incidents.
Treat Hemophilia
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Treat
HIV/AIDS
HIV is an immunodeficiency virus that attacks and suppresses the immune system making it more easy for humans to contract infections and cancer. In retrospect, HIV causes AIDS which stands for Acquired Immunodeficiency Syndrome. Although this is still in research, with gene therapy, scientists are able to disable the gene that makes the HIV entryway OR add a gene to help T-cells recognize and kill HIV.
Treat
HIV/AIDS
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How does bioinformatics play a role in gene therapy?
In the past, there have been many problems facing gene therapy. However, with greater understanding of the human genome, gene therapy is able to evolve with the help of bioinformatics. This eventually makes gene therapy become a virtual reality. Take for example, adenoviruses used in human gene therapy as gene transfer vectors. Combined with the use of bioinformatics, it allows for the safe and rational approach to designing vectors.
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Bioinformatics Aid in gene therapy:
Implications for Gene Therapy and Vaccine Vector Development
HAdV-4 is an adenovirus that is also a reemerging viral pathogenic agent that is linked in epidemic outbreaks of respiratory disease. Genomic and bioinformatic analysis of a prototype nucleotide genome suggests a closer phylogenetic relationship with the chimpanzee adenoviruses rather than with other human viruses. This ultimately suggests a recent origin of the HAdV-4 adenovirus through human interaction with animals - from chimpanzees to humans. Bioinformatics analysis also suggest implications for current interest in sing chimpanzee adenoviruses in the development of vectors for human gene therapy and for DNA-Based vaccines.
Bioinformatics Help Reasearchers Find New Uses for Old Drug
By linking cancer gene expression patterns with drug activity, a research team has found a possible cancer therapy hidden in an antimicrobial agent. Using a novel bioinformatics approach, a team of researchers used a collection of gene expression data from human cancer cells treated with hundreds of small molecule drugs to help in the treatment of patients with advanced kidney cancer.
Bioinformatic Strategies to Rapidly Characterize Retroviral Integration Sites of Gene Therapy Vectors
Two bioinformatic programs: IntegrationSeq and IntegrationMap were created in order to allow large-scale and standardized comparison of insertion sites of viral vectors. IntergrationSeq is able to trim sequences and when combined with IntegrationMap, valid integration sequences are further processed. Detailed information about whether inegrations are located in or close to genes, name of gene, the exact location in the transcriptional units, and the distance form the transcription start site to the integration. Using IntegrationSeq and IntegrationMap, a validated, fast and standardized high-throughput analysis of insertion sites can be achieved for the first time.
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