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Category Archives: Gene Therapy Trials

Challenges in Gene Therapy – Learn Genetics

Posted: November 4, 2015 at 12:41 pm

HOME Gene Therapy Challenges in Gene Therapy? Continue reading

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Gene Therapy and Children – KidsHealth

Posted: August 21, 2015 at 1:45 pm

Gene therapy carries the promise of cures for many diseases and for types of medical treatment that didn’t seem possible until recently. With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and cancer, gene therapy is a potential medical miracle-worker. But what about gene therapy for children? Continue reading

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Gene Therapy – Genetics Home Reference

Posted: August 15, 2015 at 7:41 am

Please choose from the following list of questions for information about gene therapy, an experimental technique that uses genetic material to treat or prevent disease. On this page: 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 Continue reading

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Prof Krystof Bankiewicz. Overview of AADC Deficiency – AAV2-hAADC Gene Therapy Trials. – Video

Posted: December 27, 2014 at 5:42 pm

Prof Krystof Bankiewicz. Continue reading

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With Collaboration, Scientists Test Gene Therapy for 'Bubble Boy Disease'

Posted: October 21, 2014 at 1:45 am

A new variation of gene therapy raises hopes for a safe and effective long-term treatment for X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening heritable disorder. The research was produced by a collaborative research team from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, along with other institutions participating in an international clinical trial that involved boys from the United States and France Continue reading

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New Initiatives to Protect Participants in Gene Therapy Trials

Posted: May 8, 2014 at 7:46 am

FOR IMMEDIATE RELEASE Tuesday, March 7, 2000 Contact: FDA Press Office (301) 827-6250 NIH Press Office (301) 496-5787 New Initiatives to Protect Participants in Gene Therapy Trials FDA’s clinical trials monitoring plan addresses emerging evidence that the monitoring by study sponsors of several recent gene therapy trials has been less than adequate. To buttress the rigor of the oversight, FDA will require that sponsors of gene therapy trials routinely submit their monitoring plans to the FDA. FDA will review these monitoring plans and seek modifications as warranted to improve the quality of monitoring Continue reading

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Patient Receives First-Ever Gene Therapy for Parkinson's at …

Posted: December 26, 2013 at 11:48 pm

Patient Receives First-Ever Gene Therapy for Parkinson’s at NewYork-Presbyterian Hospital/Weill Cornell Medical Center Historic Procedure Also Marks First In Vivo Gene Therapy in Adult Brain NEW YORK (Aug 20, 2003) Surgeons at NewYork-Presbyterian/Weill Cornell performed the world’s first gene therapy for Parkinson’s disease on a 55-year-old New York man on Monday, August 18. The historic surgery, which also marked the first-ever in vivo gene therapy in the brain for an adult neurological disease, was part of a Phase I clinical trial approved by the Food and Drug Administration in October 2002 Continue reading

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Gene Therapy Clinical Trials Databases

Posted: November 28, 2013 at 5:45 pm

Wiley database on Gene Therapy Trials Worldwide The Journal of Gene Medicine clinical trial site presenting charts and tables showing the number of approved, ongoing or completed clinical trials worldwide. Data is available for: Continents and countries where trials are being performed; Indications addressed; Vectors used; Gene types transferred; Phases of clinical trials; Number of trial approved/initiated 1989-2007. A searchable database is also present with detailed information on individual trials. Continue reading

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Will Jones – Training for run for Cystic Fibrosis Trust Rocky Training Sequence – Video

Posted: November 1, 2012 at 9:46 pm

Will Jones – Training for run for Cystic Fibrosis Trust Rocky Training Sequence My name is William Jones. I'm 10 years old and have got cystic fibrosis. Continue reading

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Jonathan on BBC Breakfast

Posted: April 3, 2011 at 6:19 am

Interview on BBC Breakfast with Professor Robin Ali about Gene Therapy Trials for Eye Conditions Continue reading

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