AAVLife (2014 ). AAVLife raises $12 million in series A financing to advance gene therapy for Friedreich's Ataxia. Available at www.versantventures.com/wp-content/uploads/2014/04/AAVLife-Raises.pdf (accessed May7, 2014).
2.
Avalanche Biotechnologies (2014). Avalanche Biotechnologies secures $55 million in oversubscribed series B financing. Available at www.avalanchebiotech.com/pdf/Avalanche-Biotechnologies-Series-B.pdf (accessed May6, 2014).
3.
Celladon (2014). Celladon receives breakthrough therapy designation from FDA for Mydicar®, novel, first-in-class therapy in development to treat heart failure. Available at http://ir.celladon.net/releasedetail.cfm?ReleaseID=839474 (accessed May7, 2014).
4.
CongL., RanF.A., CoxD., et al. (2013). Multiplex genome engineering using CRISPR/Cas systems. Science, 339, 819–823. Also available at www.sciencemag.org/content/339/6121/819 (accessed May6, 2014).
5.
Editas Medicine (2014). Broad Institute awarded first patent for engineered CRISPR-Cas9 system. Available at www.editasmedicine.com/documents/Broad%20Institute%20awarded%20first%20patent%20for%20engineered%20CRISPR.pdf (accessed May6, 2014).
6.
Massachusetts Institute of Technology (MIT; 2014). Erasing a genetic mutation. Available at http://newsoffice.mit.edu/2014/erasing-genetic-mutation (accessed May6, 2014).
7.
Oxford BioMedica (2014a). Oxford BioMedica completes patient recruitment into RetinoStat® Phase I trial. Available at http://www.oxfordbiomedica.co.uk/press-releases/oxford-biomedica-completes-patient-recruitment-into-retinostat-r-phase-i-trial/ (accessed May9, 2014).
8.
Oxford BioMedica (2014b). Oxford BioMedica provides update on Retino-Stat® programme. Available at www.oxfordbiomedica.co.uk/press-releases/oxford-biomedica-provides-update-on-retinostat-r-programme/ (accessed May9, 2014).
9.
Oxford BioMedica (2014c). Oxford BioMedica receives Technology Strategy Board grant for OXB-102. Available at www.oxfordbiomedica.co.uk/press-releases/oxford-biomedica-receives-technology-strategy-board-grant-for-oxb-102/ (accessed May9, 2014).
10.
PalfiS., GurruchagaJ.M., RalphG.S., et al. (2014). Long-term safety and tolerability of ProSavin. The Lancet, 383, 1138–1146. Also available at http://cc.bingj.com/cache.aspx?q=Long-term+safety+and+tolerability+of+ProSavin&d=4806937298272943&mkt=en-US&setlang=en-US&w=-0ewEVPtkOqJ8TeXUCVMHZwMbjNdv2Ko (accessed May9, 2014).
11.
PerdominiM., BelbellaaB., MonassierL., et al. (2014). Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia. Nature Med. [Epub ahead of print]; doi:10.1038/nm.3510. Available at www.nature.com/nm/journal/vaop/ncurrent/abs/nm.3510.html (accessed April9, 2014)
12.
Regeneron Pharmaceuticals and Avalanche Biotechnologies (2014). Regeneron and Avalanche Biotechnologies announce collaboration to develop next-generation gene therapy products in ophthalmology. Available at http://investor.regeneron.com/releasedetail.cfm?ReleaseID=845170 (accessed May6, 2014).
13.
ReGenX Biosciences and AAVLife (2014). ReGenX Biosciences enters into license agreement with AAVLife for development of treatments for Friedrich's Ataxia using NAV® vectors. Available at http://regenxbio.com/resources/uploads/general_files/AAVLifePressReleaseFinal.pdf (accessed May7, 2014).
14.
YinH., XueW., ChenS., et al. (2014). Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nature Bio. [Epub ahead of print]; doi:10.1038/nbt.2884. Available at www.nature.com/nbt/journal/vaop/ncurrent/abs/nbt.2884.html (accessed May6, 2014).