Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
- Submitting institution
-
University of Keele
- Unit of assessment
- 12 - Engineering
- Output identifier
- 440
- Type
- D - Journal article
- DOI
-
10.1073/pnas.1605731113
- Title of journal
- Proceedings of the National Academy of Sciences
- Article number
- -
- First page
- 10962
- Volume
- 113
- Issue
- 39
- ISSN
- 0027-8424
- Open access status
- Compliant
- Month of publication
- September
- Year of publication
- 2016
- URL
-
https://www.pnas.org/content/113/39/10962
- Supplementary information
-
-
- Request cross-referral to
- -
- Output has been delayed by COVID-19
- No
- COVID-19 affected output statement
- -
- Forensic science
- No
- Criminology
- No
- Interdisciplinary
- No
- Number of additional authors
-
11
- Research group(s)
-
-
- Proposed double-weighted
- No
- Reserve for an output with double weighting
- No
- Additional information
- This study demonstrated the potential of cell-penetrating peptides (CPPs) to deliver gene-targeting therapies efficiently and non-invasively. The work was funded by MRC (MR/L013142/1, U105178803 & G0900887), Bethlehem Griffith Research Foundation (BGRF1501), and SMA UK. The CPP technology and chemistry within this study are contained within patent (WO/2018/150196), underpin the PepGen spin out (www.pepgen.com), and contributed to the MDUK PhD (RA3/3031/1) of Dr Katharina Meijboom, currently a post-doc at the University of Massachusetts Medical School. The study was selected for the Naked Scientist podcast (https://www.thenakedscientists.com/podcasts/naked-scientists-podcast/little-light-relief). Follow-on funding from MRC-DPFS funding (MR/R025312/1) was obtained to pursue the development of CPPs.
- Author contribution statement
- -
- Non-English
- No
- English abstract
- -
