NGF and BDNF in pediatrics syndromes

Area of Science:

• Neuroscience
• Developmental Biology
• Genetics

Background:

• Neurotrophins (NTs), including nerve growth factor (NGF) and brain-derived neurotrophic factor (BDNF), are vital for neuronal development, function, and survival in both central and peripheral nervous systems.
• These factors play critical roles from early developmental stages onwards.

Purpose of the Study:

• To review and summarize existing evidence on the role of NTs in rare and uncommon pediatric human diseases characterized by neurodevelopmental changes.
• To explore the association between NTs and various pathological manifestations in these conditions.

Main Methods:

• Literature review and evidence synthesis.
• Analysis of studies linking neurotrophin alterations to specific pediatric diseases and their associated symptoms.

Main Results:

• Numerous rare pediatric diseases, including chronic granulomatous disease, hereditary sensory and autonomic neuropathy, Duchenne muscular dystrophy, Bardet-Biedl syndrome, Angelman syndrome, fragile X syndrome, trisomy 16, Williams-Beuren syndrome, Prader-Willi syndrome, WAGR syndrome, fetal alcohol spectrum disorders, Down syndrome, and Klinefelter Syndrome, have been analyzed.
• NT alterations are associated with a wide spectrum of pathologies such as cognitive defects, behavioral abnormalities, epilepsy, obesity, tumorigenesis, and muscle-skeletal, immune, bowel, pain sensibility, and cilia-related disorders.

Conclusions:

• Neurotrophin alterations are implicated in the pathogenesis of a diverse range of rare pediatric neurodevelopmental disorders.
• Further research is essential to elucidate the precise therapeutic potential of NTs for these often intractable conditions.