First-pass extracted concept

Disease-modifying neurotrophic-factor gene therapy for Parkinson's disease

Candidate: concept label1 source documents3 linked claims

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Extracted Explainers

What the tool is doing

This review frames neurotrophic-factor transgenes as disease-modifying PD gene therapy candidates.

Source 1DOI PubMed

What problem it solves

They are intended to address the unmet goal of slowing disease progression rather than only improving symptoms.

Source 1DOI PubMed

What it does not solve

The abstract indicates that clinical proof of efficacy was lacking at the time, with neurturin trials failing their primary endpoint.

Source 1DOI PubMed

Alternatives

The review contrasts these disease-modifying approaches with symptomatic dopamine- or GABA-synthesis strategies.

Source 1DOI PubMed

Evidence Snippets

As for disease modifying targets that theoretically offer the possibility of slowing the progression of disease, several neurotrophic factors show encouraging results in preclinical models

Evidence 1Source 1DOI PubMedprovenance

Supporting Sources

Source 1review2018Journal of Parkinson s DiseaseDOI PubMed

1 evidence snippet 3 linked claims Claim 1 Claim 2 Claim 3

Linked Claims

Claim 1clinical outcome summarysupports2018Source 1DOI PubMed

At the time of the review, neurturin was the only neurotrophic factor tested in Parkinson's disease clinical trials and no such trial had met its primary endpoint.

Claim 2preclinical supportsupports2018Source 1DOI PubMed

Several neurotrophic-factor disease-modifying targets showed encouraging results in preclinical Parkinson's disease models.

Claim 3review summarysupports2018Source 1DOI PubMed

Parkinson's disease gene therapy has included both non-disease-modifying and disease-modifying transgenes in animal and human studies.