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Category Archives: Gene Therapy

3rd International Conference and Exhibition on Cell & Gene Therapy – Video

Posted: Published on June 14th, 2014

3rd International Conference and Exhibition on Cell Gene Therapy 3rd International Conference and Exhibition on Cell Gene Therapy to be held during October 27-29, 2014 at Las Vegas, USA. Cell Therapy conference is a rema... By: omicsgroup incorporation … Continue reading

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Attack of the B – Team EP8 – Drowning for Science! – Video

Posted: Published on June 12th, 2014

Attack of the B - Team EP8 - Drowning for Science! Attack of the B-Team Modded Minecraft on the Seed Team server with JSeed47 and friends. A little gene therapy and testing death for science... Modpack: http://www.technicpack.net/attack-of-the-bt... By: DerpTater … Continue reading

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Bluebird, Agios: Preview of Next Week’s Important Pipeline Updates

Posted: Published on June 12th, 2014

NEW YORK (TheStreet) -- Two members of the Biotech IPO Class of 2013 -- Bluebird Bio (BLUE) and Agios Pharmaceuticals (AGIO) -- will deliver important pipeline updates at the European Hematology Association (EHA) Congress on Sat. June 14. Let's preview both presentations, starting with Bluebird and its LentiGlobin gene therapy for beta-thalassemia. Lenti what for beta who? LentiGlobin is gene therapy, meaning it insertsa fully functional gene for human beta-globin into the patient's own hematopoietic stem cells. The theory behind gene therapy is relatively simple: For diseases caused by single, malfunctioning or missing gene, effective treatment or even a complete cure can be achieved (theoretically) by replacing the defective gene with one that is fully functional. Gene therapy sounds simple but the execution is obviously a lot more complicated. I won't get into the weeds explaining the process by which BlueBird produces and delivers LentiGlobin except to say a working gene for human beta-globin would allows beta-thalassemia (B-Thal) patients to produce functional beta-globin -- the largest component of hemoglobin, which carries oxygen in red blood cells. B-Thal patients suffer from anemia and iron overload. There is no currently approved cure or effective treatment, so patients require regular blood transfusions to … Continue reading

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Gene therapy combined with IMRT reduces rate of positive prostate biopsy after treatment for intermediate-risk …

Posted: Published on June 4th, 2014

Combining oncolytic adenovirus-mediated cytotoxic gene therapy (OAMCGT) with intensity modulated radiation therapy (IMRT) reduces the risk of having a positive prostate biopsy two years after treatment in intermediate-risk prostate cancer without affecting patients' quality of life, according to a study published in the June 1, 2014 edition of the International Journal of Radiation Oncology Biology Physics (Red Journal), the official scientific journal of the American Society for Radiation Oncology (ASTRO). Previous prospective clinical trials in prostate cancer have shown that increasing the standard radiation dose of 70 Gy by 10 to 15 percent improves the biochemical disease-free survival in some prognostic risk groups; however, more than 25 percent of men with intermediate- or high-risk disease develop prostate-specific antigen (PSA) progression within 10 years, suggesting that radiation doses higher than 80 Gy may be necessary. This prospective randomized phase II trial examines the use of OAMCGT to improve the effectiveness of IMRT without increasing the radiation dose in intermediate-risk prostate cancer. Based on encouraging results from a prior phase I trial, 44 patients were enrolled in this randomized phase II trial from January 2008 to July 2010. Patients were randomized to receive either OAMCGT with IMRT (21) or IMRT alone (23), … Continue reading

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Gene therapy combined with IMRT found to reduce recurrence for select prostate cancer patients

Posted: Published on June 4th, 2014

PUBLIC RELEASE DATE: 2-Jun-2014 Contact: Brittany Ashcroft press@astro.org 703-839-7336 American Society for Radiation Oncology Fairfax, Va., June 2, 2014Combining oncolytic adenovirus-mediated cytotoxic gene therapy (OAMCGT) with intensity modulated radiation therapy (IMRT) reduces the risk of having a positive prostate biopsy two years after treatment in intermediate-risk prostate cancer without affecting patients' quality of life, according to a study published in the June 1, 2014 edition of the International Journal of Radiation Oncology Biology Physics (Red Journal), the official scientific journal of the American Society for Radiation Oncology (ASTRO). Previous prospective clinical trials in prostate cancer have shown that increasing the standard radiation dose of 70 Gy by 10 to 15 percent improves the biochemical disease-free survival in some prognostic risk groups; however, more than 25 percent of men with intermediate- or high-risk disease develop prostate-specific antigen (PSA) progression within 10 years, suggesting that radiation doses higher than 80 Gy may be necessary. This prospective randomized phase II trial examines the use of OAMCGT to improve the effectiveness of IMRT without increasing the radiation dose in intermediate-risk prostate cancer. Based on encouraging results from a prior phase I trial, 44 patients were enrolled in this randomized phase II trial from January … Continue reading

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Gene Therapy Combined with IMRT Found to Reduce the Rate of Positive Prostate Biopsy After Treatment for Intermediate …

Posted: Published on June 4th, 2014

Contact Information Available for logged-in reporters only Newswise Fairfax, Va., June 2, 2014Combining oncolytic adenovirus-mediated cytotoxic gene therapy (OAMCGT) with intensity modulated radiation therapy (IMRT) reduces the risk of having a positive prostate biopsy two years after treatment in intermediate-risk prostate cancer without affecting patients quality of life, according to a study published in the June 1, 2014 edition of the International Journal of Radiation Oncology Biology Physics (Red Journal), the official scientific journal of the American Society for Radiation Oncology (ASTRO). Previous prospective clinical trials in prostate cancer have shown that increasing the standard radiation dose of 70 Gy by 10 to 15 percent improves the biochemical disease-free survival in some prognostic risk groups; however, more than 25 percent of men with intermediate- or high-risk disease develop prostate-specific antigen (PSA) progression within 10 years, suggesting that radiation doses higher than 80 Gy may be necessary. This prospective randomized phase II trial examines the use of OAMCGT to improve the effectiveness of IMRT without increasing the radiation dose in intermediate-risk prostate cancer. Based on encouraging results from a prior phase I trial, 44 patients were enrolled in this randomized phase II trial from January 2008 to July 2010. Patients were … Continue reading

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BEYOND THE BUBBLE: 10 Years of Gene Therapy – Video

Posted: Published on May 31st, 2014

BEYOND THE BUBBLE: 10 Years of Gene Therapy 10 minute documentary/feature created as my end of year project for my Masters course in TV Journalism at Nottingham Trent University. Made by David Sykes on... By: David Sykes … Continue reading

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Genetic Researching: Gene Therapy – Video

Posted: Published on May 31st, 2014

Genetic Researching: Gene Therapy Bio Video. By: Ali Banach … Continue reading

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Genome Editing to Reverse Bubble Boy Syndrome

Posted: Published on May 31st, 2014

Researchers used an emerging technique to correct the gene behind a fatal immune system disorder in an infant. A new kind of gene therapy which involves editing, rather than replacing, faulty genes in sick people, is being used experimentally in patients. The latest report shows how scientists can correct a broken gene as it sits in the patients genome. How the health of the patient, a 4-month old infant, will change is yet to be reported. Genome editing technology is considered a promising new tool for curing disease. For decades, gene therapy has meant that a virus delivers a functional copy of a gene that is dysfunctional in a patient. The dysfunctional copy remains and the therapeutic version typically remains separate from the rest of the genome. The technology has drawbacks. First, by sitting outside of the genome, the activity of therapeutic gene isnt regulated properly. In some cases, the therapeutic copy is delivered by a retrovirus the plunks the new gene down near randomly in the patients genome, which risks disrupting another gene, potentially causing cells to turn cancerous. Second, some diseases, such as Huntingtons, cant be treated this way because the broken copy of the gene causes harm. … Continue reading

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Spark Therapeutics late-stage gene therapy for inherited blindness takes a $73M step forward

Posted: Published on May 31st, 2014

AChildrens Hospital of Philadelphia spinout with the potential to be the first to win U.S. FDA approval of a gene therapy is now funded through the rest of development of its lead treatment for inherited blindness. Spark Therapeutics is in the middle of a Phase 3 clinical trial that will determine whether the gene therapy its developed can successfully restore some vision to people with rare degenerative eye diseases caused by mutations in the RPE65 gene. Theres currently no treatmentavailable for these diseases. To allow Spark to continue that study and prepare for commercialization, the company has just closed an oversubscribed $72.8 million Series B financing. Investors including Sofinnova Ventures, Brookside, Deerfield, Rock Springs Capital, T. Rowe Price, Wellington and CHOP participated. Developed at CHOPs Center for Cellular and Molecular Therapeutics, the therapy uses a neutralized virus as a vehicle to deliver a function gene to targeted cells in the eye. Once there, it enables production of a critical protein thats missing as a result of a mutation and causes vision loss. In earlier studies, the company reported that some children who were nearly blind as the result of a RPE65 mutation were able to recognize faces and walk without … Continue reading

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