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@ARTICLE{HassaniNia:490287,
author = {Hassani Nia, Fatemeh and Woike, Daniel and Bento, Isabel
and Niebling, Stephan and Tibbe, Debora and Schulz, Kristina
and Hirnet, Daniela and Skiba, Matilda and Hönck,
Hans-Hinrich and Veith, Katharina and Günther, Christian
and Scholz, Tasja and Bierhals, Tatjana and Driemeyer,
Joenna and Bend, Renee and Failla, Antonio Virgilio and
Lohr, Christian and Alai, Maria Garcia and Kreienkamp,
Hans-Jürgen},
title = {{S}tructural deficits in key domains of {S}hank2 lead to
alterations in postsynaptic nanoclusters and to a
neurodevelopmental disorder in humans},
journal = {Molecular psychiatry},
volume = {29},
number = {6},
issn = {1359-4184},
address = {London},
publisher = {Macmillan},
reportid = {PUBDB-2022-07620},
pages = {1683 - 1697},
year = {2022},
abstract = {Postsynaptic scaffold proteins such as Shank, PSD-95, Homer
and SAPAP/GKAP family members establish the postsynaptic
density of glutamatergic synapses through a dense network of
molecular interactions. Mutations in SHANK genes are
associated with neurodevelopmental disorders including
autism and intellectual disability. However, no SHANK
missense mutations have been described which interfere with
the key functions of Shank proteins believed to be central
for synapse formation, such as GKAP binding via the PDZ
domain, or Zn2+-dependent multimerization of the SAM domain.
We identify two individuals with a neurodevelopmental
disorder carrying de novo missense mutations in SHANK2. The
p.G643R variant distorts the binding pocket for GKAP in the
Shank2 PDZ domain and prevents interaction with Thr(−2) in
the canonical PDZ ligand motif of GKAP. The p.L1800W variant
severely delays the kinetics of Zn2+-dependent
polymerization of the Shank2-SAM domain. Structural analysis
shows that Trp1800 dislodges one histidine crucial for Zn2+
binding. The resulting conformational changes block the
stacking of helical polymers of SAM domains into sheets
through side-by-side contacts, which is a hallmark of Shank
proteins, thereby disrupting the highly cooperative assembly
process induced by Zn2+. Both variants reduce the
postsynaptic targeting of Shank2 in primary cultured neurons
and alter glutamatergic synaptic transmission.
Super-resolution microscopy shows that both mutants
interfere with the formation of postsynaptic nanoclusters.
Our data indicate that both the PDZ- and the SAM-mediated
interactions of Shank2 contribute to the compaction of
postsynaptic protein complexes into nanoclusters, and that
deficiencies in this process interfere with normal brain
development in humans.},
cin = {EMBL-User / EMBL / CSSB-EMBL / CSSB-CF-SPC},
ddc = {610},
cid = {I:(DE-H253)EMBL-User-20120814 / I:(DE-H253)EMBL-20120731 /
I:(DE-H253)CSSB-EMBL-20141216 /
I:(DE-H253)CSSB-CF-SPC-20210520},
pnm = {6G3 - PETRA III (DESY) (POF4-6G3)},
pid = {G:(DE-HGF)POF4-6G3},
experiment = {EXP:(DE-H253)P-P14-20150101},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:36450866},
UT = {WOS:000912572000001},
doi = {10.1038/s41380-022-01882-3},
url = {https://bib-pubdb1.desy.de/record/490287},
}