Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

@inproceedings{Shabanpoor2017IdentificationOA,
  title={Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy},
  author={Fazel Shabanpoor and Suzan M Hammond and Frank Abendroth and Gareth Hazell and Matthew J. Wood and Michael J. Gait},
  booktitle={Nucleic acid therapeutics},
  year={2017}
}
Splice-switching antisense oligonucleotides are emerging treatments for neuromuscular diseases, with several splice-switching oligonucleotides (SSOs) currently undergoing clinical trials such as for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). However, the development of systemically delivered antisense therapeutics has been hampered by poor tissue penetration and cellular uptake, including crossing of the blood-brain barrier (BBB) to reach targets in the central nervous… CONTINUE READING
5
Twitter Mentions

References

Publications referenced by this paper.
SHOWING 1-10 OF 80 REFERENCES

Stimulated endocytosis in penetratin uptake: effect of arginine and lysine.

  • Biochemical and biophysical research communications
  • 2008
VIEW 4 EXCERPTS
HIGHLY INFLUENTIAL

Mechanism of uptake of C105Y, a novel cell-penetrating peptide.

  • The Journal of biological chemistry
  • 2006
VIEW 6 EXCERPTS
HIGHLY INFLUENTIAL

Biogen completes rolling submission of new drug application to FDA for Nusinersen as a treatment for spinal muscular atrophy

M Hache, KJ Swoboda, +4 authors KM Bishop
  • 2016

Similar Papers

Loading similar papers…