Gene therapy is a 'disruptive science' ready for commercial development

January 24, 2012
Human Gene Therapy, the official journal of nine international societies, is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. The journal is expanding to 18 issues, to include part B HGT Methods. Credit: ©2012, Mary Ann Liebert Inc., publishers

The time for commercial development of gene therapy has come. Patients with diseases treatable and curable with gene therapy deserve access to the technology, which has demonstrated both its effectiveness and feasibility, says James Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy in a provocative commentary and accompanying videocast. Human Gene Therapy and Human Gene Therapy (HGT) Methods are peer-reviewed journals published by Mary Ann Liebert, Inc..

Until recently, has been reserved for severe diseases with few treatment options. But the recent report of its successful use to treat B, which would offer patients a therapeutic alternative that could replace the need for regular, lifelong protein replacement infusions, has brought gene therapy to the forefront as a technology capable of competing with and disrupting traditional forms of treatment. Although gene therapy for hemophilia B is still in early-stage clinical testing, a similar approach is in development to treat hemophilia A, and together these life-threatening diseases represent a $6.5 billion market for current protein replacement therapies.

The technical feasibility of gene therapy "has been established in multiple diseases and with different technology platforms," says Dr. Wilson, in the Commentary "It's Time for Gene Therapy to Get Disruptive!" He predicts that "2012 will usher in an era of commercial development of gene therapy that, although likely to begin slowly, will quickly gather momentum."

"The scientific community has been promising for years that disruptive change would follow from investments in , such as the doubling of NIH and the sequencing of the ," says Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Dean, Provost, and Executive Deputy Chancellor, Gene Therapy Center, and Departments of Pediatrics and Microbiology & Physiologic Systems, University of Massachusetts Medical School. "Dr. Wilson's comments strike at the heart of fulfilling that promise. Now that the technology is working, the next phase is to develop commercially viable models for gene therapy in the health care marketplace, so that these therapies can be delivered to the patients who need them."

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not rated yet Jan 24, 2012
Its good and effective but who knows what will happen in future.

Deletion & addition of codons might make the gene unstable and patient will be prone to cancer later on his/her life.

But I think, as nature does it billions of times (for e.g. while cell division, growth, sperm & ova creation, fertilization etc) it should be made very safe.

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