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GDNF gene therapy
- Development stage
- Unknown
- Lead developer
- AskBio
- Modality
- Lipid-mediated Delivery → Gene Addition/Replacement → Gene Therapies, Electrical: Electroporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Mechanical: Microinjection, microfluidics, gene gun → Physical Methods → Gene Addition/Replacement → Gene Therapies, Acoustic/Optical: Sonoporation, laser photoporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Pressure-driven: Hydrodynamic injection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Magnetic: Magnetofection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intracerebral, Intraputaminal
01
Overview
GDNF gene therapy is an investigational treatment that delivers the human glial cell line-derived neurotrophic factor (GDNF) gene to targeted brain regions using viral or non-viral vectors. The most studied approach uses adeno-associated viral vector serotype 2 (AAV2-GDNF or AB-1005), which enables stable and continuous expression of GDNF in localized areas after direct neurosurgical injection. GDNF is a protein that promotes the survival and regeneration of dopaminergic neurons, which are progressively lost in Parkinson’s disease. By increasing local production of GDNF, this therapy aims to protect and restore dopamine-producing cells, potentially slowing disease progression rather than just alleviating symptoms[1][4][8]. Non-viral delivery methods such as Trojan horse liposomes have also been explored for systemic administration[5]. The primary indication under investigation is Parkinson’s disease.
- Other names
- glial cell line-derived neurotrophic factor gene therapyGDNF gene transfer
02
Targets
GFRα1 (Glial cell line-derived neurotrophic factor receptor alpha 1)NCAM1 (Neural cell adhesion molecule 1)RET (Rearranged during transfection receptor tyrosine kinase)GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)
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