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SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy
Winer L, Srinivasan D, Chun S, Lacomis D, Jaffa M, Fagan A, Holtzman DM, Wancewicz E et al.. JAMA neurology, 2013-02-01
Topic: Brain proteins, microglia and lab models
Background Therapies designed to decrease the level of SOD1 are currently in a clinical trial for patients with superoxide dismutase (SOD1)-linked familial amyotrophic lateral sclerosis (ALS). Objective To determine whether the SOD1 protein in cerebral spinal fluid (CSF) may be a pharmacodynamic marker for antisense oligonucleotide therapy and a disease marker for ALS. Design Antisense oligonucleotides targeting human SOD1 were administered to rats expressing SOD1G93A.
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
APA 7th
Winer, L., Srinivasan, D., Chun, S., Lacomis, D., Jaffa, M., Fagan, A., et al. (2013). SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. JAMA neurology. https://doi.org/10.1001/jamaneurol.2013.593
Vancouver
Winer L, Srinivasan D, Chun S, Lacomis D, Jaffa M, Fagan A, et al. SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. JAMA neurology. 2013. doi:10.1001/jamaneurol.2013.593. PMID: 23147550.
BibTeX
@article{winer2013-23147550,
author = {Winer, L. and Srinivasan, D. and Chun, S. and Lacomis, D. and Jaffa, M. and Fagan, A. and Holtzman, D. M. and Wancewicz, E. and others},
title = {{SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy}},
journal = {JAMA neurology},
year = {2013},
doi = {10.1001/jamaneurol.2013.593},
pmid = {23147550},
url = {https://doi.org/10.1001/jamaneurol.2013.593}
}
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Key findings
- Objective To determine whether the SOD1 protein in cerebral spinal fluid (CSF) may be a pharmacodynamic marker for antisense oligonucleotide therapy and a disease marker for ALS.
- Design Antisense oligonucleotides targeting human SOD1 were administered to rats expressing SOD1G93A.
- The human SOD1 protein levels were measured in the rats' brain and CSF samples.
- In human CSF samples, the following proteins were measured: SOD1, tau, phosphorylated tau, VILIP-1, and YKL-40.
Why it matters
This study examined whether SOD1 protein in cerebrospinal fluid could serve as a marker for antisense oligonucleotide therapy and for ALS, using rats and human samples. It concerns ALS rather than Alzheimer disease and does not test any Alzheimer treatment, cure, or prevention.
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Study details
- Effect size7.1%; 5.7%
- Evidence strengthLow
How this study scored 46/100
Published in a leading specialty journal. Scored zero for study design, sample size, studied in people and randomised / blinded because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 9 / 10 | Tier 2 — leading specialty journal (JAMA neurology) |
| Journal's citation record | 2 / 4 | JAMA Neurology: journal h-index 235 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 1 / 5 | Rated “low” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 1 / 5 | 2013 — within 20 year(s) of the newest study here |
| Sample size | 0 / 4 | Participant count not stated — scores zero (not stated in the record — scored 0) |
| Effect reported | 2 / 3 | Effect reported as a number, without an interval or test |
| Studied in people | 0 / 3 | Not stated whether this was studied in people — scores zero (not stated in the record — scored 0) |
| Randomised / blinded | 0 / 2 | No randomisation or blinding stated — scores zero (not stated in the record — scored 0) |
| Times cited | 5 / 7 | Cited 111 times (OpenAlex, 2026-10-10) |
| Citations per year | 2 / 5 | 7.4 citations a year over 15 year(s) since 2013 |
| Impact for its field and year | 3 / 6 | Cited 4.3× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 1 / 6 | Cited by 2 other studies in this library |
| Still being cited | 2 / 4 | Cited 18 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 3 / 4 | Last-listed author Timothy M. Miller: h-index 60 (OpenAlex, whole career) |
| Institution's track record | 2 / 3 | Massachusetts General Hospital: institution h-index 2,057 (OpenAlex) |
| Multi-centre collaboration | 1 / 3 | OpenAlex lists 3 institution(s) in 1 country |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 23147550) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 46 / 100 | — |
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Read the original
- DOI: 10.1001/jamaneurol.2013.593
- PubMed: 23147550
- Original source →
- Free full text: PubMed Central →
Full text: free to read — OpenAlex lists this copy as free to read: the link above (checked 6 Feb 1020).
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