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gamma-aminobutyric acid + glutamic acid decarboxylase 65
- Development stage
- Unknown
- Lead developer
- Diamyd Medical
- Modality
- Small Molecules, Recombinant Proteins and Enzymes
- Administration
- Oral, Subcutaneous
01
Overview
This is a combination of gamma-aminobutyric acid (GABA), the primary inhibitory neurotransmitter in the central nervous system, and glutamic acid decarboxylase 65 (GAD65), an enzyme responsible for catalyzing the conversion of glutamate to GABA. The combination has been studied as a therapeutic approach to delay progression of type 1 diabetes mellitus (T1DM) by modulating immune responses and preserving pancreatic β-cell function. Oral administration of GABA together with injections of recombinant human GAD65 aims to enhance endogenous inhibitory signaling and induce immune tolerance against β-cell antigens. This strategy is under investigation primarily in pediatric populations with newly diagnosed T1DM[1][4]. Mechanistically, exogenous GABA may directly suppress immune-mediated β-cell destruction while recombinant GAD65 acts as an autoantigen to promote regulatory immune responses.
- Other names
- GABA + GAD65gamma-aminobutyric acid + GAD65GABA plus glutamic acid decarboxylase 65
02
Targets
GAT (GABA transporter family)GABRR (GABA-A receptor subunit rho)GABA-B (Gamma-aminobutyric acid receptor type B)Glutamate decarboxylase 65-specific T-cell receptor (GAD65-specific TCR)
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