Filmmaker Rob Whittlesey’s PBS-aired documentary examines the huge potential of using gene therapy in the treatment of acute medical conditions, including the rare genetic retinal disease Leber congenital amaurosis, cystic fibrosis, Duchenne muscular dystrophy, and the neurologic disease known as fatal familial insomnia. The Gene Doctors follows patients who undergo gene therapy treatments while also describing in accessible terms how these treatments are supposed to work. Perhaps the most fascinating story here involves treating Leber congenital amaurosis using Spark Therapeutics’ Luxturna, which received U.S. Food and Drug Administration approval in December 2017. In this process, one injection into each eye introduces a good copy of the problematic gene that could lead to blindness. Here, a canine test patient responds successfully to the treatment, as does a young teenage girl who has a fondness for puzzle solving. The medical professionals behind these advances have been working for years to perfect these treatments, although it will still be some time before they become low-cost, commonplace solutions available to a wider population. A compelling film that posits an encouraging future for eradicating illnesses once seen as an early death sentence, this is highly recommended. Aud: H, C, P. (P. Hall)
The Gene Doctors
(2017) 55 min. DVD: $24.99 ($54.99 w/PPR). PBS Video. SDH captioned. ISBN: 978-1-5317-0365-3. Volume 33, Issue 3
The Gene Doctors
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