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Lysosomal dysfunction in Down Syndrome and Alzheimer mouse models is caused by selective v-ATPase inhibition by Tyr 682 phosphorylated APP βCTF
Im E, Jiang Y, Stavrides P, Darji S, Erdjument-Bromage H, Neubert TA, Choi JY, Wegiel J et al.. Science advances, 2022-06-04
Topic: Brain proteins, microglia and lab models
Lysosome dysfunction arises early and propels Alzheimer’s Disease (AD). Herein, we show that amyloid precursor protein (APP), linked to early-onset AD in Down Syndrome (DS), acts directly via its β-C-terminal fragment (βCTF) to disrupt lysosomal v-ATPase and acidification. In human DS fibroblasts, the phosphorylated 682 YENPTY internalization motif of APP-βCTF binds selectively within a pocket of the v-ATPase V0a1 subunit cytoplasmic domain and competitively inhibits association of the V1 subcomplex of v-ATPase, thereby reducing its activity.
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APA 7th
Im, E., Jiang, Y., Stavrides, P., Darji, S., Erdjument-Bromage, H., Neubert, T. A., et al. (2022). Lysosomal dysfunction in Down Syndrome and Alzheimer mouse models is caused by selective v-ATPase inhibition by Tyr 682 phosphorylated APP βCTF. Science advances. https://doi.org/10.1101/2022.06.02.494546
Vancouver
Im E, Jiang Y, Stavrides P, Darji S, Erdjument-Bromage H, Neubert TA, et al. Lysosomal dysfunction in Down Syndrome and Alzheimer mouse models is caused by selective v-ATPase inhibition by Tyr 682 phosphorylated APP βCTF. Science advances. 2022. doi:10.1101/2022.06.02.494546.
BibTeX
@article{im2022-4ba4a0f3,
author = {Im, E. and Jiang, Y. and Stavrides, P. and Darji, S. and Erdjument-Bromage, H. and Neubert, T. A. and Choi, J. Y. and Wegiel, J. and others},
title = {{Lysosomal dysfunction in Down Syndrome and Alzheimer mouse models is caused by selective v-ATPase inhibition by Tyr 682 phosphorylated APP βCTF}},
journal = {Science advances},
year = {2022},
doi = {10.1101/2022.06.02.494546},
url = {https://doi.org/10.1101/2022.06.02.494546}
}
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Key findings
- Herein, we show that amyloid precursor protein (APP), linked to early-onset AD in Down Syndrome (DS), acts directly via its β-C-terminal fragment (βCTF) to disrupt lysosomal v-ATPase and acidification.
- In human DS fibroblasts, the phosphorylated 682 YENPTY internalization motif of APP-βCTF binds selectively within a pocket of the v-ATPase V0a1 subunit cytoplasmic domain and competitively inhibits association of the V1 subcomplex of v-ATPase, thereby reducing its activity.
- Lowering APP-βCTF Tyr 682 phosphorylation restores v-ATPase and lysosome function in DS fibroblasts and in vivo in brains of DS model mice.
- Notably, lowering APP-βCTF Tyr 682 phosphorylation below normal constitutive levels boosts v-ATPase assembly and activity, suggesting that v-ATPase may also be modulated tonically by phospho-APP-βCTF.
Why it matters
In human Down syndrome cells, a fragment of the amyloid precursor protein was found to inhibit a lysosomal pump and reduce lysosomal acidification. This is laboratory cell research identifying a possible mechanism, not a treatment, cure or prevention for Alzheimer's disease.
Study details
- Evidence strengthLow
How this study scored 36/100
Published in a peer-reviewed journal. Scored zero for study design, sample size, effect reported 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 23 of the 64 points we could judge it on, which is 36 out of 100.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (Science advances) |
| 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 | 3 / 5 | 2022 — within 6 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 | Laboratory or animal study (title names “mouse”) — scores zero |
| 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 | 2 / 2 | Indexed in PubMed (PMID 37494443) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Points | 23 / 64 | Scored on 12 of 20 parts. We have no data for the rest, so they are left out: 23 of the 64 points we could judge it on is 36 out of 100. |
| Total | 36 / 100 | — |
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