Crystallographic and Modeling Study of the Human PPA1 (Inorganic
Pyrophosphatase 1): a Potential Anti-cancer Drug Target
Abstract
Inorganic pyrophosphatases (PPases) catalyze the hydrolysis of
pyrophosphate to phosphates. PPases play essential roles in growth and
development, and are found in all kingdoms of life. Human possess two
PPases, PPA1 and PPA2. PPA1 is present in all tissues, acting largely as
a housekeeping enzyme. Besides pyrophosphate hydrolysis, PPA1 can also
directly dephosphorylate phosphorylated JNK1. Upregulated expression of
PPA1 has been linked to many human malignant tumors. PPA1 knockdown
induces apoptosis and decreases proliferation. PPA1 is emerging as a
potential prognostic biomarker and target for anti-cancer drug
development. In spite of the biological and physiopathological
importance of PPA1, there is no detailed study on the structure and
catalytic mechanisms of mammalian origin PPases. Here we report the
crystal structure of human PPA1 at a resolution of 2.4 Å. We also
carried out modeling studies of PPA1 in complex with JNK1 derived
phosphor-peptides. The monomeric protein fold of PPA1 is similar to
those found in other family I PPases. PPA1 forms a dimeric structure
that should be conserved in animal and fungal PPases. Analysis of the
PPA1 structure and comparison with available structures of PPases from
lower organisms suggest that PPA1 has a largely pre-organized and
relatively rigid active site for pyrophosphate hydrolysis. Results from
the modeling study indicate the active site of PPA1 has the potential to
accommodate double-phosphorylated peptides derived from JNK1. In short,
results from the study provides new insights into the mechanisms of
human PPA1 and basis for structure-based anti-cancer drug developments
using PPA1 as the target.