Category Archives: Gene Therapy

JFK - Gene Therapy for Brain Tumors By: JFK Medical Center … Continue reading →

Mark Hamalainen: Gene Therapy Mark's talk at Future Friday 04/04/14. By: Julia Bossmann … Continue reading →

Gene Therapy Narrator: In Boston, scientists are working at the frontier of genetic research in an attempt to cure Macular Degeneration, the leading cause of blindness in... By: American Macular Degeneration Foundation … Continue reading →

The Harvard-MIT genomic science institute stays mute on how it will assert control over the tools expected to speed cures and change gene therapy. One of the most important genetic technologies developed in recent years is now patented, and researchers are wondering what they will and wont be allowed to do with the powerful method for editing the genome. On Tuesday, the Broad Institute of MIT and Harvard announced that it had been granted a patent covering the components and methodology for CRISPRa new way of making precise, targeted changes to the genome of a cell or an organism. CRISPR could revolutionize biomedical research by giving scientists a more efficient way of re-creating disease-related mutations in lab animals and cultured cells; it may also yield an unprecedented way of treating disease (see Genome Surgery). The patent, issued just six months after its application was filed, covers a modified version of the CRISPR-Cas9 system found naturally in bacteria, which microbes use to defend themselves against viruses. The patent also covers methods for designing and using CRISPRs molecular components. The inventor listed on the patent is Feng Zhang, an MIT researcher and core faculty member of the Broad. Zhang was an MIT … Continue reading →

Ex vivo gene therapy This gene therapy lecture explains the process of ex vivo gene therapy and the use of ex vivo gene therapy in treating diseases. For more information, log on... By: Suman Bhattacharjee … Continue reading →

In vivo gene therapy This gene therapy lecture explains the process of in vivo gene therapy and the use of in vivo gene therapy in treating diseases. For more information, log on... By: Suman Bhattacharjee … Continue reading →

Gene therapy basics This gene therapy lecture explains the basics of gene therapy in treatment of human diseases. For more information, log on to- http://shomusbiology.weebly.co... By: Suman Bhattacharjee … Continue reading →

Gene Therapy PSA Dan Lesser Period 2 Ms. Mattairs PSA Bioethics PSA By Dan Lesser Period:2 I do not own this song or any footage. By: Dan Lesser … Continue reading →

Gene therapy is the use of DNA as a drug to treat disease by delivering therapeutic DNA into a patient's cells. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease. Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID.[1] Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.[2] Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes since 2006 have bolstered new optimism in the promise of gene therapy. These … Continue reading →

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or knocking out, a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures. MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy. Educational resources related to gene therapy are available from GeneEd. The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy and a discussion of several diseases for which … Continue reading →