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Toward an NGF-based therapy for Rett syndrome
Giulia Borgonovo1, Alexia Tiberi1, Simona Capsoni1,2
1Bio@SNS Laboratory of Biology, Scuola Normale Superiore, Pisa, Italy.
Frontiers in Neuroscience
|May 11, 2026
Summary
Nerve growth factor (NGF) shows promise for Rett syndrome (RTT) treatment. Intranasal NGF variants improved RTT mouse models
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Rett syndrome (RTT) is a severe neurodevelopmental disorder linked to MECP2 gene mutations.
- Current treatments offer limited relief, necessitating novel therapeutic strategies.
- Nerve growth factor (NGF) exhibits neurotrophic and immunomodulatory potential.
Purpose of the Study:
- To review Rett syndrome and the role of growth factors.
- To highlight evidence for NGF-based therapies in RTT models.
- To analyze intranasal NGF administration in Mecp2-mutant mice.
Main Methods:
- Review of existing literature on RTT and NGF.
- Analysis of two studies using intranasal recombinant human NGF (rhNGF) and a modified variant (hNGFp) in Mecp2-mutant mice.
- Evaluation of behavioral, mitochondrial, and neuroinflammatory outcomes.
Main Results:
- Both rhNGF and hNGFp improved cognitive and motor symptoms in RTT mice.
- rhNGF primarily restored mitochondrial function; hNGFp modulated neuroinflammation via microglia.
- Intranasal administration was effective, safe, and enhanced translational potential.
Conclusions:
- NGF-based strategies show therapeutic potential for RTT.
- NGF may act via dual mechanisms: supporting energy homeostasis and regulating immune responses.
- Intranasal delivery offers a promising route for NGF therapy in RTT.
