Abstract
The armamentarium of modern genetics and gene therapy has recently expanded with the rapid development of the tools of “genome editing,” including zinc-finger nucleases, TALENs, and CRISPR/Cas9 techniques that allow exquisitely targeted sequence modifications. An alluring application of such technology is somatic cell genome editing for the prevention and therapy of genetic disease. Most scientific, ethics, and policy agencies support somatic cell applications of genome editing; the gene therapy community has understandably taken deep interest because we represent the major biomedical conduit through which the technology will eventually be translated to clinical application.
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Schedule a callMore From: Molecular therapy : the journal of the American Society of Gene Therapy
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