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   <ui>gb-spotlight-20020109-01</ui>
   <ji>GBJ</ji>
   <fm>
      <dochead>Research news</dochead>
      <bibl>
         <title>
            <p>SMaRT correction</p>
         </title>
         <aug>
            <au id="A1">
               <snm>Weitzman</snm>
               <mi>B</mi>
               <fnm>Jonathan</fnm>
               <email>jonathanweitzman@hotmail.com</email>
            </au>
         </aug>
         <source>Genome Biology</source>
         <issn>1465-6906</issn>
         <pubdate>2002</pubdate>
         <volume>3</volume>
         <fpage>spotlight-20020109-01</fpage>
         <xrefbib>
            <pubid idtype="doi">10.1186/gb-spotlight-20020109-01</pubid>
         </xrefbib>
      </bibl>
      <history>
         <pub>
            <date>
               <day>09</day>
               <month>01</month>
               <year>2002</year>
            </date>
         </pub>
      </history>
      <cpyrt>
         <year>2002</year>
         <collab>BioMed Central Ltd</collab>
      </cpyrt>
      <shortabs>
         <p>Gene therapists have developed a RNA-mediated technique for correcting gene defects that cause cystic fibrosis.</p>
      </shortabs>
   </fm>
   <meta>
      <classifications>
         <classification type="STATUS">Archive</classification>
      </classifications>
   </meta>
   <bdy>
      <sec>
         <st>
            <p/>
         </st>
         <p>A technique called <abbr bid="B1">SMaRT</abbr> (spliceosome-mediated RNA <it>Trans</it>-splicing) has been developed to generate functionally corrected mRNA transcripts and proteins in patients with genetic diseases. In the January issue of <abbr bid="B2"><it>Nature Biotechnology</it></abbr>, Liu <it>et al.</it> describe the use of SMaRT technology to correct endogenous &#916;F508 mutations in the cystic fibrosis transmembrane conductance regulator (<it>CFTR</it>) gene (<it>Nature</it><it>Biotechnology</it> 2002, <b>20:</b>47-52). They constructed recombinant adenoviral vectors containing CFTR exons 10-24 and a <it>trans</it>-splicing domain. These could partially restore CFTR chloride ion conductance in &#916;F508 airway epithelial cells <it>in vitro</it>. The SMaRT vectors resulted in the expression of corrected CFTR mRNA and protein. Liu <it>et al.</it> also demonstrated functional correction <it>in vivo</it> using a human bronchial xenograft model. These results show the potential feasibility of using SMaRT technology to correct, rather than replace, defective genes.</p>
      </sec>
   </bdy>
   <bm>
      <refgrp>
         <bibl id="B1">
            <note>Spliceosome-mediated RNA trans-splicing as a tool for gene therapy.</note>
            <xrefbib>
               <pubid idtype="pmpid" link="fulltext">10096291</pubid>
            </xrefbib>
         </bibl>
         <bibl id="B2">
            <url>http://biotech.nature.com</url>
            <note>
               <it>Nature</it>
               <it>Biotechnology</it>
            </note>
         </bibl>
      </refgrp>
   </bm>
</art>
