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Crispr/cas9 como alternativa terapêutica para pacientes com doença de Alzheimer: uma revisão integrativa
Manuela dos Santos Albuquerque, Carolina Melo de Souza. Revista Neurociências, 2026-01-30
Topic: Amyloid, tau and blood-test diagnosis
Introducción. La enfermedad de Alzheimer (EA) es una condición neurodegenerativa progresiva caracterizada por el deterioro cognitivo, la acumulación de beta-amiloide y la neuroinflamación. La tecnología CRISPR/Cas9 ha emergido como una herramienta prometedora para investigar y, potencialmente, tratar la EA, permitiendo ediciones precisas del genoma y la modulación de la expresión génica. Este estudio analizó los avances recientes en el uso de CRISPR/Cas9 para comprender los mecanismos moleculares de la EA y explorar enfoques terapéuticos. Método. Se realizó una revisión integrativa de la literatura, seleccionando estudios publicados en los últimos 10 años en portugués, inglés y español. La búsqueda incluyó artículos que investigan la manipulación de genes relacionados con la EA utilizando CRISPR/Cas9, con énfasis en estudios preclínicos y experimentales in vivo. Resultados. Los hallazgos destacan la capacidad de CRISPR/Cas9 para crear modelos experimentales que reproducen mutaciones genéticas humanas, como en los genes TREM2 y PSEN1, permitiendo investigar alteraciones sinápticas, inflamación cerebral y depósito de beta-amiloide. Además, la corrección de mutaciones como PSEN1 M146L demostró eficacia en la reducción de beta-amiloide y la restauración de funciones celulares. Sin embargo, desafíos como mutaciones off-target y limitaciones en modelos experimentales resaltan la necesidad de mejoras. El desarrollo de gRNAs específicos y el uso de organoides cerebrales se identifican como alternativas prometedoras. Conclusión. El sistema CRISPR/Cas9 ofrece un potencial terapéutico significativo para la EA, con implicaciones para tratar otras enfermedades neurodegenerativas. No obstante, son indispensables avances técnicos y estudios más profundos para una efectiva traslación clínica.
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
APA 7th
Albuquerque, M. d. S., & de Souza, C. M. (2026). Crispr/cas9 como alternativa terapêutica para pacientes com doença de Alzheimer: uma revisão integrativa. Revista Neurociências. https://doi.org/10.34024/rnc.2026.v34.19837
Vancouver
Albuquerque MdS, de Souza CM. Crispr/cas9 como alternativa terapêutica para pacientes com doença de Alzheimer: uma revisão integrativa. Revista Neurociências. 2026. doi:10.34024/rnc.2026.v34.19837.
BibTeX
@article{albuquerque2026-b001c286,
author = {Albuquerque, Manuela dos Santos and de Souza, Carolina Melo},
title = {{Crispr/cas9 como alternativa terapêutica para pacientes com doença de Alzheimer: uma revisão integrativa}},
journal = {Revista Neurociências},
year = {2026},
doi = {10.34024/rnc.2026.v34.19837},
url = {https://doi.org/10.34024/rnc.2026.v34.19837}
}
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.
Key findings
- Además, la corrección de mutaciones como PSEN1 M146L demostró eficacia en la reducción de beta-amiloide y la restauración de funciones celulares.
- Sin embargo, desafíos como mutaciones off-target y limitaciones en modelos experimentales resaltan la necesidad de mejoras.
- El desarrollo de gRNAs específicos y el uso de organoides cerebrales se identifican como alternativas prometedoras.
- El sistema CRISPR/Cas9 ofrece un potencial terapéutico significativo para la EA, con implicaciones para tratar otras enfermedades neurodegenerativas.
- No obstante, son indispensables avances técnicos y estudios más profundos para una efectiva traslación clínica.
Why it matters
This integrative review reports that CRISPR/Cas9 has been used to create experimental models with human mutations such as in TREM2 and PSEN1, and that correcting a PSEN1 M146L mutation reduced amyloid-beta and restored cellular functions. It notes challenges including off-target mutations and limitations in experimental models, so no treatment or cure in patients is established.
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Study details
- Evidence strengthLow
How this study scored 28/100
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 senior author's track record and institution's track record, so those were left out: it earned 26 of the 93 points we could judge it on, which is 28 out of 100.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (Revista Neurociências) |
| Journal's citation record | 0 / 4 | Revista Neurociências: journal h-index 21 (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 | 5 / 5 | 2026 — within 1 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 | 0 / 7 | Cited 0 times (OpenAlex, 2026-10-10) |
| Citations per year | 0 / 5 | 0.0 citations a year over 2 year(s) since 2026 |
| Impact for its field and year | 0 / 6 | Cited 0.0× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 0 / 6 | No other study in this library cites it |
| Still being cited | 0 / 4 | Cited 0 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | — / 4 | OpenAlex's profile for last-listed author Carolina Melo de Souza holds only 1 work(s), too few to judge a track record — left out of this score |
| Institution's track record | — / 3 | No institution listed in OpenAlex — left out of this score |
| Multi-centre collaboration | 0 / 3 | OpenAlex lists 0 institution(s) in 0 countries — scores zero (affiliation lists can be incomplete) |
| Indexed in PubMed | 1 / 2 | Not in PubMed, but has a DOI |
| Free to read | 2 / 2 | Free to read |
| Points | 26 / 93 | Scored on 18 of 20 parts. We have no data for the rest, so they are left out: 26 of the 93 points we could judge it on is 28 out of 100. |
| Total | 28 / 100 | — |
What each of these 20 components measures, and what this score is not →
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