Gene Therapy Helps Boys with ‘Lorenzo’s Oil’ Disease

October 5, 2017

(ABC News) – The fledgling field of gene therapy has scored another win: An experimental treatment seemed to help boys with the inherited nerve disease featured in the movie “Lorenzo’s Oil.” Fifteen of the 17 boys treated in a study had no major disability two years later — remarkable for a disease that often causes swift decline and kills within a decade.

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