Jim Wil­son of­fers a safer so­lu­tion to some of the in­dus­try's gene ther­a­py woes

Jim Wil­son has been warn­ing for a while about the dan­gers of the vi­ral vec­tors used in most gene ther­a­pies. Two years ago he walked away from Sol­id Bio­sciences as he pub­lished a pa­per show­ing the po­ten­tial ad­verse neu­ro­log­i­cal ef­fects of AAV vec­tors. Last year, he called the field’s progress on vec­tors “em­bar­rass­ing.”

Now the gene ther­a­py pi­o­neer, whose trag­ic 1999 tri­al showed the dead­ly risks of the first gen­er­a­tion of vec­tors, is of­fer­ing a so­lu­tion. On Wednes­day, he and a team of re­searchers at the Uni­ver­si­ty of Penn­syl­va­nia pub­lished a pa­per in Sci­ence Trans­la­tion­al Med­i­cine show­ing a way of mod­i­fy­ing the vec­tors so they cor­rect genes with­out hit­ting a del­i­cate clus­ters of neu­rons that, when dis­turbed, can cause dan­ger­ous side ef­fects.

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