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References
1.
Mendell
JR
,
Al-Zaidy
S
,
Shell
R
, et al. Single-dose gene-replacement therapy for spinal muscular atrophy . N Engl J Med , 2017 ; 377 :1713 –1722 .
2.
Hirsch
T
,
Rothoeft
T
,
Teig
N
, et al. Regeneration of the entire human epidermis using transgenic stem cells . Nature , 2017 ; 551 :327 –332 .
3.
Eichler
F
,
Duncan
C
,
Musolino
PL
, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy . New Engl J Med , 2017 ; 377 :1630 –1638 .
4.Novartis . Press release: Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice . https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019 (last accessed November 14 , 2017 ).
5.Gilead . Press release: Kite's Yescarta™ (Axicabtagene Ciloleucel) becomes first CAR T therapy approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy . www.gilead.com/news/press-releases/2017/10/kites-yescarta-axicabtagene-ciloleucel-becomes-first-car-t-therapy-approved-by-the-fda-for-the-treatment-of-adult-patients-with-relapsed-or-refractory-large-bcell-lymphoma-after-two-or-more-lines-of-systemic-therapy (last accessed November 14 , 2017 ).
6.Spark Therapeutics . Press release: FDA Advisory Committee unanimously recommends approval of investigational LUXTURNA™ (voretigene neparvovec) for patients with biallelic RPE65-mediated inherited retinal disease . http://ir.sparktx.com/news-releases/news-release-details/fda-advisory-committee-unanimously-recommends-approval (last accessed November 14 , 2017 ).
7.Gilead . Press release: Gilead sciences to acquire Kite Pharma for $11.9 billion . www.gilead.com/news/press-releases/2017/8/gilead-sciences-to-acquire-kite-pharma-for-119-billion (last accessed November 14 , 2017 ).
8.Dimension Therapeutics . Press release: Ultragenyx and Dimension announce expiration of Hart-Scott-Rodino waiting period for Ultragenyx's acquisition of Dimension . http://investors.dimensiontx.com/phoenix.zhtml?c=254192&p=irol-newsArticle&ID=2311718 (last accessed November 14 , 2017 ).
