Treatment approaches for hydrocephalus.
| Therapy type | Description | Current status | Advantages | Challenges | References |
|---|---|---|---|---|---|
| Surgical & shunt | VPS, ETV ± CPC, antibiotic catheters, programmable valves | Clinical use | Effective CSF diversion, reduced shunt dependence, and infection reduction | Infection, revision, and mechanical complexity | [29–34, 105, 106] |
| Pharmacological | Acetazolamide, furosemide, NKCC1 inhibitors, antifibrotics | Preclinical/Limited clinical | Non-invasive, adjunct therapy | Limited efficacy, systemic toxicity | [108–110] |
| Regenerative & cell-free | Stem cells, MSCs, EVs, ependymal reprogramming (GemC1/Mcidas) | Preclinical | Repair, neuroprotection, and restore the ependymal lining | Delivery, tumorigenicity, and scalability | [91, 113, 115] |
| Gene & molecular | CRISPR/Cas9, AAV vectors, molecular targets (NKCC1, TRPV4) | Preclinical/Experimental | Targeted therapy, disease-modifying | Immune responses, delivery challenges | [117–119] |
| Modeling platforms | Organoids, organ-on-chip, computational models | Preclinical/Regulatory | Personalized testing, patient-specific modeling | Standardization, reproducibility | [35, 39, 83, 84, 88] |
This table summarizes proven and newly emerging strategies, including surgical and shunt therapies (VPS, ETV, ETV-CPC, programmable/antibiotic valves), drug therapies (acetazolamide, furosemide, NKCC1 inhibitors, antifibrotics), regenerative and cell-free therapies (stem cells, EVs, ependymal reprogramming), molecular and gene therapies (CRISPR/Cas9, AAV vectors, ion channel targets), and bioengineered modeling platforms (organoids, organ-on-chip, computational fluid dynamics). Each entry clarifies therapeutic rationale and status. AAV: adeno-associated virus; CPC: choroid plexus cauterization; CSF: cerebrospinal fluid; ETV: endoscopic third ventriculostomy; EVs: extracellular vesicles; MSCs: mesenchymal stem cells; NKCC1: Na+-K+-2Cl– cotransporter 1; SVZ: subventricular zone; VPS: ventriculoperitoneal shunt; TRPV4: transient receptor potential vanilloid 4.
IA: Conceptualization, Methodology, Investigation, Writing—original draft, Writing—review & editing, Visualization, Project administration. The author read and approved the submitted version.
The author declares that there are no competing interests.
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This review article does not contain original datasets. All information discussed is available in the published literature and cited accordingly.
This work was supported by the Hellenic Foundation for Research and Innovation (HFRI), 3rd call for postdoctoral fellows (Research Grant No. 7371). The funder had no role in study design, data collection, data analysis, interpretation, writing of the manuscript, or the decision to submit the article for publication.
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