CRISPR technology for Parkinson’s disease: Recent advancements and ongoing challenges
Bibliographic record
Abstract
Parkinson’s disease (PD) is a neurodegenerative disorder caused by decreased dopamine, resulting in impaired motor function. Various gene editing methods are used in PD research to understand the disease’s complexity and develop treatments. With no cure and limited treatments, it is important to understand the recent advances in PD research, particularly with new gene editing technologies. Therefore, we evaluated recent advancements in gene therapy and CRISPR technology in PD research, using Pubmed to identify CRISPR use in PD research conducted within the past ten years. We compiled cell and gene therapy clinical trials for PD using clinicaltrials.gov, finding no current therapies approved for PD treatment, and CRISPR has yet to be incorporated in any clinical trials. We organized CRISPR technology used in PD research into three study types: animal models, stem cells, and cell culture. The studies reviewed involve research into genetic forms of PD and pathological hallmarks, such as α-synuclein accumulation, mitochondrial dysfunction, and cell death. Double or triple-transgenic models and induced pluripotent stem cells have been utilized more recently, contributing critical information to the understanding of PD. CRISPR is a powerful tool that has significantly advanced PD research. However, much research is still required to fully unravel the pathology and see whether CRISPR can be used in therapies to correct gene mutations and improve dysfunctional mechanisms across PD patients. Overall, CRISPR techniques for use in PD treatments are still in early development, being tested using cell and animal models that will hopefully move into clinical trials soon.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.008 | 0.006 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.002 | 0.002 |
| Science and technology studies | 0.001 | 0.002 |
| Scholarly communication | 0.003 | 0.004 |
| Open science | 0.001 | 0.002 |
| Research integrity | 0.002 | 0.003 |
| Insufficient payload (model declined to judge) | 0.004 | 0.002 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".