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In Utero gene therapy: current challenges and perspectives

21 Feb 2005

Abstract excerpt

Over the past few years, considerable progress in prenatal diagnosis and surgery combined with improvements in vector design vindicate a reappraisal of the feasibility of in utero gene therapy for serious monogenetic diseases. As adult gene therapy gathers pace, several apparent obstacles to its application as a treatment may be overcome by pre- or early postnatal treatment. This review will examine the concepts...

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Identifiers

PMID: 15851005
DOI: 10.1016/j.ymthe.2005.01.015

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