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  1. No Access

    Article

    RNAi quashes polyQ

    RNA interference in the brain inhibits neurodegeneration in a polyglutamine disease, SCA1. Is this now the way forward for the clinical treatment of certain genetic disorders (pages 816–820

    Natasha J Caplen in Nature Medicine (2004)

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    Article

    Gene silencing through RNA interference: Potential for therapeutics and functional genomics

    A major focus of biology is determining the role that specific genes play within a cell. The ability to control gene expression is a powerful tool for biologists, and methods that facilitate the expression of ...

    David O. Azorsa, Spyro Mousses, Natasha J. Caplen in Letters in Peptide Science (2003)

  3. No Access

    Chapter and Conference Paper

    Lipid Gene Trasfer, a Story of Simplicity and Complexity

    It is 10 years since the first description of the use of a positively charged lipid to deliver nucleic acids to mammalian cells (Feigner et al., 1987). In essence the methodology of liposome-mediated transfer of ...

    Natasha J. Caplen in Gene Therapy (1998)

  4. No Access

    Chapter and Conference Paper

    Gene Therapy for Cancer

    Cancer is a complex acquired disease that affects millions of individuals each year. Underlying the pathogenesis of cancer are a variety of molecular genetic abnormalities which can be inherited or environment...

    Susan A. Zullo, Natasha J. Caplen, R. Michael Blaese in Gene Therapy (1998)

  5. No Access

    Chapter and Conference Paper

    Lipid Gene Transfer and Clinical Gene Therapy

    There are several features of liposome-mediated DNA transfer that make it a viable delivery system for clinical gene therapy. 1) In contrast to viral delivery systems, contamination with exogenous protein or o...

    Natasha J. Caplen in Gene Therapy (1998)

  6. No Access

    Article

    Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

    We report the results of a double-blind, placebo-controlled trial in nine cystic fibrosis (CF) subjects receiving cationic liposome complexed with a complementary DNA encoding the CF transmembrane conductance ...

    Natasha J. Caplen, Eric W.F.W. Alton, Peter G. Mddleton, Julia R. Dorin… in Nature Medicine (1995)