GBA1

Diseases

• Parkinson disease22 trials
• Gaucher disease8 trials
• Lewy body dementia1 trial
• Multiple myeloma1 trial

The last 24 months

New trials, publications, preprints and grants on this target, month by month.

TrialsPublicationsPreprintsGrants

• Trial

  A Phase 2 study of VQ-101 in participants with Parkinson’s disease and GBA1 mutation

  13 Nov 2026· PHASE2 · NOT_YET_RECRUITING· CTIS

• Publication

  Integrative Evolutionary and Structural Analysis of GBA1 Provides Insights Into Mutation-Specific Effects Associated With Parkinson's Disease.

  1 Nov 2026· The European journal of neuroscience· PUBMED

• Publication

  Longitudinal olfactory and neuropsychological trajectories across the GBA1 spectrum.

  16 Oct 2026· Brain communications· PUBMED

• Publication

  Extended Deletion in the GBA1 Gene, Masked by Apparent p.Asn409Ser Homozygous: Diagnostic Implications and the Need for an Integrated Genetic Approach.

  15 Oct 2026· International journal of molecular sciences· PUBMED

• Publication

  GBA1 Canada initiative (G-can), a pre-competitive, open science platform for GBA1 research.

  15 Oct 2026· Journal of Parkinson's disease· PUBMED

• Publication

  Building Scalable Recruitment Models for Precision Medicine in Parkinson's Disease: Insights from GBA1 Trials.

  8 Oct 2026· Movement disorders : official journal of the Movement Disorder Society· PUBMED

• Trial

  Prevent Cognitive Decline in GBA-associated Parkinson's Disease

  1 Oct 2026· PHASE2 · RECRUITING· CTGOV

• Trial

  Parkinson's Disease in Type 1 Gaucher Disease

  21 Sept 2026· NA · RECRUITING· CTGOV

• Publication

  GBA1 in Parkinson's disease: past, present and future.

  18 Sept 2026· Brain : a journal of neurology· PUBMED

• Publication

  Early-onset parkinsonism as a presenting feature of suspected type 1 Gaucher disease with two pathogenic GBA1 variants: a case report.

  3 Sept 2026· Frontiers in neuroscience· PUBMED

• Publication

  The Effect of LRRK2 and GBA1 Mutations on Survival in Early- and Late-Onset Parkinson's Disease.

  1 Sept 2026· Movement disorders : official journal of the Movement Disorder Society· PUBMED

• Publication

  Novel splice site variants in GBA1 are associated with Gaucher disease and genotype-phenotype correlations.

  22 Aug 2026· Brain & development· PUBMED

• Publication

  Glucocerebrosidase dysfunction in GBA1 carriers: insights from blood and macrophage analyses.

  21 Aug 2026· Molecular biology reports· PUBMED

• Publication

  Pathogenic GBA1 Variants and their Interaction with the Gut-Microbiota-Brain Axis in Parkinson's Disease.

  20 Aug 2026· Movement disorders : official journal of the Movement Disorder Society· PUBMED

• Publication

  Genetic Architecture of Synaptic Failure in Dementia with Lewy Bodies: From α-Synuclein Proteoforms to GBA1-Mediated Plasticity Deficits.

  18 Aug 2026· Genes· PUBMED

• Publication

  Early-Onset Parkinson's Disease with 22q11.2 Microdeletion and Pathogenic GBA1 Variant.

  10 Aug 2026· Movement disorders clinical practice· PUBMED

• Publication

  GBA1 variants and mortality in Parkinson's disease: A systematic review and meta-analysis.

  7 Aug 2026· Journal of Parkinson's disease· PUBMED

• Publication

  Cholesterol enhances lysosome-autophagosome fusion for better α-synuclein clearance in GBA L444P-mutated Parkinson disease.

  6 Aug 2026· Cell reports· PUBMED

• Publication

  Altered synaptic and astrocytic proteins in Lewy body disorders associated with GBA mutations.

  23 Jul 2026· Brain communications· PUBMED

• Publication

  E326K GBA polymorphism is associated with clinical and pathological features of synucleinopathy in the absence of overt Parkinson's disease or Lewy body dementia.

  22 Jul 2026· Journal of Parkinson's disease· PUBMED

• Publication

  Establishment of a human induced pluripotent stem cell line (PNUSCRi003-A) from a patient with Gaucher disease carrying compound heterozygous p.Arg87Trp and p.Arg296Gln variants in the GBA1 gene.

  22 Jul 2026· Stem cell research· PUBMED

• Publication

  Association of GBA1 status and sex with depression severity and progression in Parkinson's disease.

  8 Jul 2026· NPJ Parkinson's disease· PUBMED

• Trial

  A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebral Injection of LY-N001 Injection for the Treatment of Moderate to Advanced Parkinson's Disease With GBA1 Mutations

  7 Jul 2026· EARLY_PHASE1 · NOT_YET_RECRUITING· CTGOV

• Publication

  GBA1 variants and dopaminergic imaging in essential tremor with atypical features: A comparative study with Parkinson's disease.

  6 Jul 2026· Parkinsonism & related disorders· PUBMED

• Publication

  GBA mutation exacerbates α-synuclein pathology with involvement of ROS and p38 MAPK signaling in Parkinson's disease.

  30 Jun 2026· International immunopharmacology· PUBMED

• Publication

  Ferumoxytol enhances NF-κB-responsive GBA-targeted ferroptosis gene therapy in acute myeloid leukemia.

  23 Jun 2026· Journal of nanobiotechnology· PUBMED

• Publication

  Recombinant GBA1 alleles presenting as exon-level deletions by short-read NGS in Parkinson disease: Implications for diagnostic approaches.

  19 Jun 2026· Journal of human genetics· PUBMED

• Publication

  Targeting lysosomal pH restores mitochondrial quality control in GBA1-mutant Parkinson's disease.

  17 Jun 2026· Translational neurodegeneration· PUBMED

• Publication

  Intra-CNS AAV9-GBA1 delivery yields species and route of administration differences in safety and transgene expression.

  11 Jun 2026· Molecular therapy. Advances· PUBMED

• Publication

  Lysine acetyltransferase 8-mediated histone acetylation, regulated by GBA1, is associated with lysosomal function related to α-Synuclein pathology.

  8 Jun 2026· Cell death & disease· PUBMED

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