Perinatal Gene Transfer to the Liver

  title={Perinatal Gene Transfer to the Liver},
  author={Tristan R McKay and Ahad A Rahim and Suzanne M. K. Buckley and Natalie J. Ward and Jerry K. Y. Chan and Steven J Howe and Simon N Waddington},
  booktitle={Current pharmaceutical design},
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within the first weeks or months of life is inevitable. Some diseases can be managed using conventional medicines whereas others are, as yet, untreatable. In this review we consider the application of early intervention gene therapy in neonatal and… CONTINUE READING
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Publications referenced by this paper.
Showing 1-10 of 172 references

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

  • A Nathwani, E Tuddenham, C Rosales
  • Blood
  • 2010
Highly Influential
4 Excerpts

Gene therapy for mucopolysaccharidosis

  • KP Ponder, Haskins, ME
  • Expert Opin Biol Ther 2007;
  • 2007
Highly Influential
3 Excerpts

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