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Related Concept Videos

Enzyme-linked Receptors01:00

Enzyme-linked Receptors

Enzyme-linked receptors are proteins that act as both receptor and enzyme, activating multiple intracellular signals. This is a large group of receptors that include the receptor tyrosine kinase (RTK) family. Many growth factors and hormones bind to and activate the RTKs.
Neurotrophin (NT) receptors are a family of RTKs, including trkA, trkB, and trkC (tropomyosin-related kinase) receptors. TrkA is specific for nerve growth factor (NGF), neurotrophin-6, and neurotrophin-7. TrkB binds...

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Regenerative Therapy by Suprachoroidal Cell Autograft in Dry Age-related Macular Degeneration: Preliminary In Vivo Report
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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
PubMed
Summary
This summary is machine-generated.

Nerve growth factor (NGF) shows promise for Rett syndrome (RTT) treatment. Intranasal NGF variants improved RTT mouse models

Keywords:
Rett syndromeintranasal treatmentmicrogliamitochondrianerve growth factor (NGF)

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Published on: February 17, 2015

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.