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CRISPR–Cas9–Mediated Genetic Correction for Alzheimer's Disease A Hypothetical Therapeutic Framework
Md Asif Karim, Sohana Parveen Meem, Abdul Kalam Azad. Zenodo (CERN European Organization for Nuclear Research), 2025-12-23
Topic: Amyloid, tau and blood-test diagnosis
The repeated inability of Aβ therapies to pass clinical tests in sporadic AD points to the complexity of the disease and indicates that earlier or more interventions could be necessary. Results:CRISPR/Cas9 gene editing in genes associated with AD, APP, PSEN1, BACE1, and APOE4 was very effective in mitigating β-amyloid deposition, decreasing tau hyperphosphorylation, and improving neuronal function in vivo and in vitro. Efficient delivery through viral and non-viral vectors demonstrated precise gene modification with low off-targeting, with high specificity reported in neuronal cells. Studies on functional outcomes in animal models of AD showed improved learning and memory performance, which was associated with reduced amyloid plaque burden and neuroinflammation. These findings demonstrate not only that CRISPR/Cas9 affects core pathogenic pathways but also that it is translatable to measurable cognitive enhancement, an indication of potential as a disease-modifying therapy.
Key findings
- The repeated inability of Aβ therapies to pass clinical tests in sporadic AD points to the complexity of the disease and indicates that earlier or more interventions could be necessary.
- Results:CRISPR/Cas9 gene editing in genes associated with AD, APP, PSEN1, BACE1, and APOE4 was very effective in mitigating β-amyloid deposition, decreasing tau hyperphosphorylation, and improving neuronal function in vivo and in vitro.
- Efficient delivery through viral and non-viral vectors demonstrated precise gene modification with low off-targeting, with high specificity reported in neuronal cells.
- Studies on functional outcomes in animal models of AD showed improved learning and memory performance, which was associated with reduced amyloid plaque burden and neuroinflammation.
- These findings demonstrate not only that CRISPR/Cas9 affects core pathogenic pathways but also that it is translatable to measurable cognitive enhancement, an indication of potential as a disease-modifying therapy.
Why it matters
This describes a hypothetical therapeutic framework, not a tested treatment in people, so it does not show that CRISPR/Cas9 can treat or prevent Alzheimer's in patients. It reports that gene editing reduced amyloid and tau and improved learning and memory in animal and cell models, which is early evidence only.
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Study details
- Evidence strengthLow
How this study scored 39/100
Published in a peer-reviewed journal. Scored zero for study design, sample size, effect reported, studied in people and randomised / blinded because the record does not state it. We have no data on journal's citation record, times cited, citations per year, impact for its field and year, still being cited, senior author's track record, institution's track record and multi-centre collaboration, so those were left out: it earned 25 of the 64 points we could judge it on, which is 39 out of 100.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (Zenodo (CERN European Organization for Nuclear Research)) |
| Journal's citation record | — / 4 | No journal record from OpenAlex — left out of this score |
| 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 | 4 / 5 | 2025 — within 3 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 | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| 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 | — / 7 | No citation count (not found in OpenAlex) — left out of this score |
| Citations per year | — / 5 | No citation count or no year — left out of this score |
| Impact for its field and year | — / 6 | No field-weighted citation impact (OpenAlex has none yet) — left out of this score |
| Cited by studies here | 0 / 6 | No other study in this library cites it |
| Still being cited | — / 4 | No yearly citation counts — left out of this score |
| Senior author's track record | — / 4 | Senior author's record not available — left out of this score |
| Institution's track record | — / 3 | No institution listed in OpenAlex — left out of this score |
| Multi-centre collaboration | — / 3 | Institutions not listed — left out of this score |
| Indexed in PubMed | 1 / 2 | Not in PubMed, but has a DOI |
| Free to read | 2 / 2 | Free to read |
| Points | 25 / 64 | Scored on 12 of 20 parts. We have no data for the rest, so they are left out: 25 of the 64 points we could judge it on is 39 out of 100. |
| Total | 39 / 100 | — |
What each of these 20 components measures, and what this score is not →
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Cite this study APA, Vancouver, BibTeX · download .bib or .ris
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. Check the citation against the original before you use it.
APA 7th
Karim, M. A., Meem, S. P., & Azad, A. K. (2025). CRISPR–Cas9–Mediated Genetic Correction for Alzheimer's Disease A Hypothetical Therapeutic Framework. Zenodo (CERN European Organization for Nuclear Research). https://doi.org/10.5281/zenodo.18032008
Vancouver
Karim MA, Meem SP, Azad AK. CRISPR–Cas9–Mediated Genetic Correction for Alzheimer's Disease A Hypothetical Therapeutic Framework. Zenodo (CERN European Organization for Nuclear Research). 2025. doi:10.5281/zenodo.18032008.
BibTeX
@article{karim2025-a93d1dcb,
author = {Karim, Md Asif and Meem, Sohana Parveen and Azad, Abdul Kalam},
title = {{CRISPR–Cas9–Mediated Genetic Correction for Alzheimer's Disease A Hypothetical Therapeutic Framework}},
journal = {Zenodo (CERN European Organization for Nuclear Research)},
year = {2025},
doi = {10.5281/zenodo.18032008},
url = {https://doi.org/10.5281/zenodo.18032008}
}
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