The U.S. National Library of Medicine on PubMed Health states:
“Duchenne muscular dystrophy is an inherited disorder that involves rapidly worsening muscle weakness. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition.”
Gene replacement therapy is on the forefront of potential treatment for this disease, in which a healthy copy of genetic material is delivered to the affected tissues that are plagued with the genetic mutation. A number of diseases have been positively treated with therapeutic genes in recent clinical successes, including Leber’s congenital Amaurosis and Hemophilia B.
The annual meeting of The American Society of Gene and Cell Therapy (ASGCT) will be held this year at the Philadelphia Convention Center on May 14-20th The Society, Feb., 2012 published this breaking article on important research written by Roland W. Herzog, PhD and Irene Zolotukhin, MS in Feb., 2012 and published by the ASGCT.
Among key presentations will be Translational Science Training Course – educational program that will cover a number of areas including clinical trials, regulatory affairs, biostatistics, manufacturing, preclinical studies and assays. The ASGCT has a great number of exhibitors and supporters to learn from at the event as well.
If this is a topic that interests you, yet you have little to no knowledge about it, don’t be shy about attending. While the in-depth programs are geared for junior faculty, clinical investigators and project managers, the ASGCT will hold educational sessions providing a basic overview, starting with basics and covering broad introductions about specific topics of interest.
Traveling to Philadelphia is exciting, and if this is a business-related trip for you don’t forget to book your ground transportion from the airport.
If you are attending to simplyl learn more you will want to see the sites in Philadelphia, time allowing. Remember your resource for transportation while in Philadelphia, from the airport to other local event and entertainment.
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