총정리 GBA protein & activity POSTMORTEM (continued)

L444P and R463C point mutation mouse(Ginns,) in Striatum (hetero는 안 쓰인 것 같음) CBE (Manning-Bog, midbrain) CBE (Ginns) in SN & Straitum CBE 100mg, 28 d (Rocha) → ↑ proteinase-K resistant a-syn aggregates

(Papadopoulos, 2018 #1273) CBE+A53T mice: ↑ aSyn soluble monomer in midbrain, striatum aSyn oligomer, (but no change in p-aSyn,), see additional details of Asyn different species and different regions.

(Henderson, 2020 #1233) In PFF-treated mice: CBE → ① = dopaminergic neuronal loss in SN (fig 5F), ② GCase inhibition does not induce aggregation of a-syn but rather induces mild neuronal spheroids recognized by 81A, which can cross-react with neurofilament ③ 거의, = aSyn spread (to cortex) fig 6B

Synapse

In Vitro

In VIVO (Rocha, 2015 #1253) CBE (100mg/kg, 28 d) → c1q, SNAP-25, Synaptophysin, synaptoagmin, synapsin, PSD-95

총정리 GBA protein & activity POSTMORTEM] Activity: hetero only 에선 kurzawa가 유일.

  • GlcSph
  • Blood: 에서 단독으로 본 것 없잖아!? (2017 Guedes에선 galactosylsph와의 sum), 이거 못 보나 Sato에게 물어보자.
sampleGBA proteinGBA activityGlcCerGlcSphCathepsin D proteinCathepsin D activityLIMP2LAMP2LC3
GBA+PD / Brain (Li, 2019 #745) In supplement
(Gundner et al. 2019, PMID 30236861)
(Gegg et al. 2012, PMID 23034917)
(Choi et al. 2011, PMID).
(Kurzawa-Akanbi, 2012 #577)
In supplement
(Moors, 2019 #644)
(Li, 2019 #745)
(Gundner et al. 2019, PMID 30236861)
(Gegg et al. 2012, PMID 23034917)
(Choi et al. 2011, PMID).
(Kurzawa-Akanbi, 2012 #577)
(Gegg et al. 2012, PMID 23034917 but =
(Kurzawa-Akanbi, 2012 #577) but =
In supplement
(Moors, 2019 #644)
(Kurzawa-Akanbi, 2012 #577) ↓
(Gegg et al. 2012, PMID 23034917 but =
GBA+PD / CSF Schondorf et al. 2014, PMID 24905578) In supplement
(Parnetti et al. 2017, PMID 28843015)
(Huh, 2021 #2538) PPMI, ↑ (~20%)
Ask Comp Bio
sPD / Brain
sPD / CSF(Xicoy, 2019 #2540) 2nd, Publication available in sPD(Xicoy, 2019 #2540) 2nd, Publication available in sPD(Xicoy, 2019 #2540) 2nd, Publication available in sPD

PD only / PD+ heteroz / Hetero only / GD only / PD+GD matrix

PD onlyPD+ heterozHetero onlyGD onlyPD+GD
2021 Avenali phd dissertation
(Huh, 2021 #2538) CSF
Fibroblast LIMP2, GBA activity, aSyn, gba protein, LAMP-1, Cathepsin D

Fig1) in GBA+PD (n=44): GlcCer ↑ (~18%, 0.93 vs 0.79, statistically significant, huge overlap, The median GlcCer fraction in GBA+PD patients was 0.93% (IQR, 0.67-1.20) compared to 0.79% (0.59-1.04) in HC (P = 0.0001, n=227.), no difference between mild vs severe GBA mutation
GlcCer. Spd: = vs HC
GlcSph: 논문x
Zama, 2009 #742) Fibroblast
GD type not stated,
↑ GlcCer (fig 8)
Sasagasako, 1994 #743) Fibroblast
= GlcCer,
= GlcSph (despite ↓ GBA activity)
Leyns, 2022 #2182)
Good, recent, Virginia Lee, Merck,
Postmortem brain,
DLB도 봄
In GBA+PD:
↓ GBA activity correlated with paSyn (in putamen, r=-0.26),
GlcCer. = but mildly correlated with paSyn (in cingulate and frontal)
GlcSph: = (gpa+pdd만 ↑) but correlated with paSyn (in putamen, r=0.42)
GBA activity correlated with GlcSph (in putamen, r=-0.51)
Moors, 2019 #644)
Postmortem brain,
DLB도 봄
Mol Neurobiol (2019) 56:1344-1355
Alterations in the activities of several other lysosomal enzymes were found in brain tissue and CSF of PD or DLB patients compared to controls, including β-hexosaminidase (β-Hex) [22-26, js: all sPD.
In addition, decreased protein levels have been demonstrated for various other ALP components in the PD brain [27-30, 이중 30만 GBA+PD인데 lysosome data 없는듯,,]
Fig3f) =(frontal cortex, SN) – Cathepsin D mRNA: Fig3f) =(frontal cortex, SN); Fig3f) =(frontal cortex, SN)

Cathepsin D activity: HC vs GBA+PD: not significant

Peripheral blood and GD studies

SourcePD onlyPD+ heterozHetero onlyGD onlyPD+GD
PBMC
GlcCer, GlcSph는 논문x
?, 2020 #1105) Dried blood spot
(Perkin Elmer)
GBA activity no reduction
overlap!
GBA activity 대략 60% of healthy control
?, 1997 PBMC (culture 아닌듯, CBE 줄때는 culture에)
Type 1 GD patients (n=6) GBA activity: 10% (PBMC), monocyte도 유사결과
?, 2016 Lymphocyte
[nGD] 10-30% of control (표3),
recovered by ambrosol
?, #2619) Postmortem brain,
N=21, GlcCer: =
GlcSph: not done
?, 2019 #2553) Postmortem brain,
(fig7) ↓ GBA protein (60%) & activity (40%)
Mito: ↑ VDAC, ↓ MC1 enz activity, ↑ 8-OHdG, CYCS ↑, ↓ TOMM20, ↓ TIM23, ↓ MFN1 &2, OPA1, ↓ DNM1L, ↓ FIS1
Lyso: fig8, ↑ lc3ii, ↑ lc3b, ↑ sqstm, ↑ (trend) LAMP1, ↑ SQSTM1, ↓ (p-MTOR, p-RPS6, AMBRA1, BECN1)
Cf) GbaL444P/WT mouse hippocampal neurons data available
?, 2015 Fibroblast
↓ GBA activity (js 50%, this is corrected by ambroxol, fig1), slightly ↓ GBA protein (js 10%), =Prosaposin, =saposin,)
=Cathepsin D

Uncertain Spans

locationtranscriptionuncertainty
matrix / Brain row(Choi et al. 2011, PMID).The PMID number is missing; the trailing ). is preserved.
matrix / sPD CSF(Xicoy, 2019 #2540) placement in Cathepsin D activity / LAMP2 / LC3 columnsThree identical citations appear across three columns with the same 2nd, Publication available in sPD annotation; column placement is preserved as visible.
Avenali row2021 Avenali phd dissertationthe source label and column placement are preserved as visible; the row’s exact column boundary is unclear because the upper-left cell is partly clipped.
Huh fig1 row0.93 vs 0.79 and (P = 0.0001, n=227.)reads as written; the n=227. token ends with a period whose role (decimal vs sentence) is ambiguous.
Lower postmortem rows?, 2020 #1105), ?, 1997, ?, 2016, ?, #2619), ?, 2019 #2553), ?, 2015Author names in the leftmost column are clipped or omitted (rendered as ?); EndNote-style identifiers are preserved as visible.