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BDNF-overexpressing human striatal progenitor cells
- Development stage
- Unknown
- Lead developer
- Stanford University
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intracerebral, Intrastriatal
01
Overview
BDNF-overexpressing human striatal progenitor cells are a type of **cell therapy** engineered from human pluripotent stem cells (hPSCs) to **constitutively express brain-derived neurotrophic factor (BDNF)** for therapeutic purposes. These cells are designed for direct transplantation into the striatum. The overexpression of BDNF is intended to restore neurotrophic support in conditions like Huntington's disease (HD), where striatal BDNF levels are pathologically reduced. The cells are further modified to include inducible safety switches that can be activated (e.g., by rapamycin) to eliminate the cells if safety concerns arise. In preclinical studies, intrastriatal transplantation of these BDNF-overexpressing striatal progenitors resulted in improvement of motor and cognitive deficits, reduction of mutant huntingtin protein aggregates, and other neuroprotective effects in HD animal models[2][4]. This approach aims at combining the benefits of regenerative cell therapy with targeted trophic factor delivery, while also addressing biosafety concerns.
- Other names
- BDNF-overexpressing human striatal progenitor cellsSTRpcs overexpressing BDNFBDNF-STRpcs
02
Targets
NTRK2 (Tropomyosin-related kinase receptor type B)
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