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Last update 08 May 2025

GANAB

Last update 08 May 2025

Basic Info

Synonyms

Alpha-glucosidase 2, G2AN, GANAB

+ [6]

Introduction

Catalytic subunit of glucosidase II that cleaves sequentially the 2 innermost alpha-1,3-linked glucose residues from the Glc(2)Man(9)GlcNAc(2) oligosaccharide precursor of immature glycoproteins (PubMed:10929008). Required for PKD1/Polycystin-1 and PKD2/Polycystin-2 maturation and localization to the cell surface and cilia (PubMed:27259053).

Drugs associated with GANAB

Prodrugs of IHVR-19029

Target

GANAB x α-glucosidase

Mechanism

GANAB inhibitors [+1]

Active Org.

Baruch S. Blumberg Institute

Originator Org.

Baruch S. Blumberg Institute

Active Indication

Hemorrhagic Fever, Ebola [+1]

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

100 Clinical Results associated with GANAB

100 Translational Medicine associated with GANAB

0 Patents (Medical) associated with GANAB

174

Literatures (Medical) associated with GANAB

08 Apr 2025·Clinical Kidney Journal

The spectrum of diseases, genetic landscape and new mutation sites of hereditary cystic kidney disease

Article

Author: Li, Shuang ; Wang, Peng ; Xie, Yuansheng ; Li, Qinggang ; Guo, Wenkai ; Bi, Jingru ; Ji, Pengcheng

ABSTRACTBackgroundCystic kidney disease is common. Beyond autosomal dominant polycystic kidney disease (ADPKD), knowledge of other hereditary forms of cystic kidney disease remains limited. This study aimed to retrospectively analyse 702 patients with multiple renal cysts from the Chinese PLA General Hospital (September 2015–December 2023).MethodsPatients suspected of having hereditary cystic kidney disease underwent next-generation sequencing (NGS) and subsequent bioinformatics analysis. Variations were assessed for pathogenicity in accordance with the American College of Medical Genetics and Genomics (ACMG) guidelines. Moreover, the ClinVar and Mastermind databases were used to identify novel mutation sites. Statistical analysis was performed using SPSS 25.0 software.ResultsOf 702 patients, 96 (13.7%) lacked gene mutations associated with cystic kidney disease. In contrast, 606 patients (86.3%) were found to have gene mutations associated with renal cyst phenotypes, involving 23 unique mutated genes. Among these, mutations in 158 patients were categorized as variants of uncertain significance. The remaining 448 patients harboured mutations predicted by the ACMG guidelines to be pathogenic or likely pathogenic, enabling a diagnosis of hereditary cystic kidney disease. These mutations were linked to seven diseases and 10 genes. The most common was ADPKD [434 cases (96.9%)], followed by autosomal dominant tubulointerstitial kidney disease [ADTKD; six cases (1.3%)], autosomal recessive polycystic kidney disease [ARPKD; five cases (1.1%)] and tuberous sclerosis complex [two cases (0.4%)]. One case each was found for autosomal dominant polycystic liver disease, COL4A1-related disease and IFT140-related disease. The mutated genes included PKD1, PKD2, GANAB, HNF1B, REN, PKHD1, ALG8, IFT140, COL4A1 and TSC2. Moreover, 63 novel pathogenic or likely pathogenic variants were identified.ConclusionIn this study we identified 23 mutated genes linked to renal cyst phenotypes, 10 of which had pathogenic or likely pathogenic variants. These findings facilitated the diagnosis of seven hereditary cystic kidney diseases, including ADPKD, ADTKD, ARPKD and others. Furthermore, 63 novel pathogenic or likely pathogenic variants were identified.

01 Mar 2025·Comparative Biochemistry and Physiology Part C: Toxicology & Pharmacology

Endocytosis, endoplasmic reticulum, actin cytoskeleton affected in tilapia liver under polystyrene microplastics and BDE153 acute co-exposure

Article

Author: Hu, Jiawen ; Zheng, Yao ; Li, Jiajia ; Xu, Gangchun ; Sun, Yi ; Zhu, Haojun

Studies showed that contaminants adhered to the surface of nano-polystyrene microplastics (NPs) have a toxicological effect. Juveniles tilapia were dispersed into four groups: the control group A, 75 nm NPs exposed group B, 5 ng·L^-1 2,2',4,4',5,5'-hexabromodiphenyl ether group C (BDE₁₅₃), and 5 ng·L^-1 BDE₁₅₃ + 75 nm MPs group D, and acutely exposed for 2, 4 and 8 days. The hepatic histopathological change, enzymatic activities, transcriptomics, and proteomics, have been performed in tilapia. The results showed that the enzymatic activities of anti-oxidative (ROS, SOD, EROD), energy (ATP), lipid metabolism (TC, TG, FAS, LPL, ACC), pro-inflammatory (TNFα, IL-1β) and apoptosis (caspase 3) significantly increased at 2 d in BDE₁₅₃ and the combined group and together in BDE₁₅₃ group at 8 d. Histological slice showed displaced nucleus by BDE₁₅₃ exposure and vacuoles appeared in the combined groups. KEGG results revealed that pathways associated with endocytosis, protein processing in endoplasmic reticulum and regulation of actin cytoskeleton were significantly enriched. The selected genes associated with neurocentral development (ganab, diaph3/baiap2a/ddost decreased and increased), lipid metabolism (ldlrap1a decreased, stt3b increased), energy (agap2 decreased, uggt1 increased) were affected under co-exposure, and fibronectin significantly increased via proteome. Our study indicated that endocytosis, protein processing in endoplasmic reticulum, regulation of actin cytoskeleton were affected in tilapia liver under NPs and BDE₁₅₃ co-exposure.

01 Mar 2025·Molecular & Cellular Proteomics

Deleterious knock-outs in the HLA class I antigen processing and presentation machinery induce distinct changes in the immunopeptidome

Article

Author: Bassani-Sternberg, Michal ; Maschke, Clélia ; Spaapen, Robbert ; Michaux, Justine ; Pak, HuiSong ; Wessling, Laura ; Verkerk, Tamara ; Shapiro, Ilja E

The human leukocyte antigen (HLA) processing and presentation machinery (APPM) is altered in various diseases and in response to drug treatments. Defects in the machinery may change presentation levels or alter the repertoire of presented peptides, globally or in an HLA allele restricted manner, with direct implications for adaptive immunity. In this study, we investigated the immunopeptidome landscape across a panel of isogenic HAP1 cell line clones each with a knock-out of a single gene encoding a key protein in the APPM, including B2M, TAP1, TAP2, TAPBP, IRF2, PDIA3, ERAP1, GANAB, SPPL3, CANX, and CALR. We applied immunopeptidomic and proteomic to assess the successful gene knock-outs on the protein level, to understand how these proteins participate in antigen presentation, and to contextualize protein expression and antigen presentation. We validated the absence of the knocked-out proteins in the respective samples and found that knocking-out an APPM component leads to the loss of peptide subsets that are normally presented on the control wild type cells. We assessed the immunopeptidomes qualitatively and quantitatively, considering factors like peptide diversity, peptide length distribution, and binding affinity to the endogenously expressed HLA alleles in HAP1 cells. We demonstrated a prominent HLA allele-specific alterations in several knock-out conditions. The absence of CALR, CANX, and TAP1 led to significant changes in HLA allele-specific presentation levels. Overall, this work represents the first systematic analysis of how the absence of individual APPM components, knocked out in a single cell line under controlled conditions, affects the peptidome. This approach could facilitate the creation of predictive tools capable of prioritizing HLA-bound peptides likely to be presented when presentation defects occur, such as in cancer and viral infections.

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GANAB

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