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25. K. Weintraub, “New gene therapy shows promise for patients with sickle cell disease,” WBUR, March 8, 2019, https://www.wbur.org/commonhealth/2019/03/08/gene-therapy-sickle-cell.
26. Sharon Begley, “NIH and Gates Foundation launch effort to bring genetic cures for HIV, sickle cell disease to world’s poor,” STAT, October 23, 2019, https://www.statnews.com/2019/10/23/nih-gates-foundation-genetic-cures-hiv-sickle-cell/.
27. Sean Nolan, “Unite to Cure,” The Vatican, April 26, 2018.
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30. Larry Luxner, “With Zolgensma’s approval, debate shifts to pricing and availability of world’s costliest drug,” SMA News Today, May 29, 2019, https://smanewstoday.com/2019/05/29/zolgensma-approval-shifts-debate-pricing-availability-worlds-costliest-drug/l.
31. Nathan Yates, “I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point,” STAT, May 31, 2019, https://www.statnews.com/2019/05/31/spinal-muscular-atrophy-zolgensma-price-critics/.
32. E. Mamcarz et al., “Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1,” New England Journal of Medicine 380, (2019); 1525–1534, https://www.nejm.org/doi/full/10.1056/NEJMoa1815408.
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35. George Church, interview, Boston, August 3, 2019.
36. Sarah Boseley, “Dismay at lottery for $2.1m drug to treat children with muscle-wasting disease,” Guardian, December 20, 2019, https://www.theguardian.com/society/2019/dec/20/lottery-prize-zolgensma-drug-zolgensma-children-muscle-wasting-disease.
37. F. W. Twort, “An investigation on the nature of ultra-microscopic viruses,” Lancet 186, (1915): 1241–1243.
38. A. Dublanchet, “The epic of phage therapy,” Canadian Journal of Infectious Diseases and Medical Microbiology 18, (2007): 15–18, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2542892/.
39. Richard Martin, “How ravenous Soviet viruses will save the world,” WIRED, October 1, 2003, https://www.wired.com/2003/10/phages/.
40. M. Rosen, “Phage therapy treats patient with drug-resistant bacterial infection,” Howard Hughes Medical Institute, May 8, 2019, https://www.hhmi.org/news/phage-therapy-treats-patient-with-drug-resistant-bacterial-infection.
41. Julianna LeMieux, “Phage therapy win: Mycobacterium infection halted,” Genetic Engineering & Biotechnology News, May 8, 2019, https://www.genengnews.com/insights/phage-therapy-win-mycobacterium-infection-halted/.
42. Ben Fidler, “How an Ohio kids’ hospital quietly became ground zero for gene therapy,” Xconomy, April 15, 2019, https://xconomy.com/national/2019/04/15/how-an-ohio-kids-hospital-quietly-became-ground-zero-for-gene-therapy/.
43. Ryan Cross, “The redemption of James Wilson,” Chemical & Engineering News, September 2019, https://cen.acs.org/business/The-redemption-of-James-Wilson-gene-therapy-pioneer/97/i36.
44. Ibid.
45. C. Hinderer et al., “Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN,” Human Gene Therapy 29, (2018): 285–298.
46. Ben Fidler, “Two patients die in now-halted study of Audentes gene therapy,” BioPharma Dive, June 26, 2020, https://www.biopharmadive.com/news/audentes-gene-therapy-patient-deaths/580670/
47. Nicole Paulk, “Gene Therapy: It’s Time to Talk about High-Dose AAV,” Genetic Engineering & Biotechnology News, July 7, 2020, https://www.genengnews.com/topics/genome-editing/gene-therapy-its-time-to-talk-about-high-dose-aav/.
Chapter 12: Fix You
1. Rebecca Robbins, “Billionaire Sean Parker is nerding out on cancer research. Science has never seen anyone quite like him,” STAT, July 9, 2019, https://www.statnews.com/2019/07/09/sean-parker-cancer-research-science/.
2. F. Baylis and M. McLeod, “First-in-human Phase 1 CRISPR gene editing cancer trials: Are we ready?,” Current Gene Therapy 17, (2017): 309–319, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5769084/.