Abstract: The disclosure relates to methods of using block copolymer nanoparticles for in vivo therapeutic delivery, and methods therefor. More particularly, the invention relates to methods of using polymer nanoparticles for delivering nucleic acids for treating NF1.

Abstract: The present disclosure provides methods and compositions for the treatment of NF-1 and NF-1 mediated conditions. The present disclosure further provides for methods of exon skipping and exon retention and compositions for use in such methods. Such methods of exon skipping and exon retention may be used in the methods of treatment discussed herein. The present disclosure further provides new therapeutic compounds, particularly oligonucleotides, including antisense oligonucleotides, for use in the methods described herein.