Evidence map›Paper›PMID 41171314›Full record

ReviewNeurogenetics2025

CRISPR-based gene therapy for huntington's disease: current advances and future prospects.

Amna Zaheer, Noha Mohamed AboQuella, Al-Hassan Soliman Wadan, Hager Adel Saad, Danisha Kumar, Shaliza Panjwani, Shree Rath, Syed Ijlal Ahmed

Abstract readReview
PubMed Publisher
In one paragraph

Review in Neurogenetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Amna ZaheerDepartment of Internal Medicine, Liaquat National Hospital and Medical College, Karachi, Pakistan. zaheeramna31@gmail.com.
Noha Mohamed AboQuellaDepartment of Neuroscience, Vrije Universiteit Amsterdam, De Boelelaan 1105, Amsterdam, 1081 HV, The Netherlands.
Al-Hassan Soliman WadanOral Biology Department, Faculty of Dentistry, Galala University, Galala Plateau, Suez Governorate, Attaka, 15888, Egypt.
Hager Adel SaadDepartment of pharmacy and biotechnology, German University in Cairo (GUC), New Cairo, Cairo, Egypt.
Danisha KumarDepartment of Internal Medicine, Dow University of Health Sciences, Karachi, Pakistan.
Shaliza PanjwaniDepartment of Internal Medicine, Dow University of Health Sciences, Karachi, Pakistan.
Shree RathAll India Institute of Medical Sciences, Bhubaneswar, India.
Syed Ijlal AhmedDepartment of Neurology, SSM Health Saint Louis University, St. Louis, MO, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

CRISPR-Cas9 technology offers transformative potential in treating Huntington's Disease (HD) by directly addressing its genetic root causes. This manuscript explores the pathophysiological mechanisms of HD, characterized by toxic mutant huntingtin (mHTT) protein resulting from expanded CAG repeats in the HTT gene, and the challenges posed by current therapeutic limitations. We comprehensively review the mechanisms of CRISPR-based therapeutic strategies, including excision of expanded repeats, allele-specific targeting, and epigenome editing, highlighting their efficacy in preclinical studies using animal models and human iPSCs. Delivery methods, such as viral and non-viral vectors, are analysed for their role in optimizing therapeutic outcomes while minimizing off-target effects and immune responses. Ethical and safety considerations, especially regarding precision and long-term impacts, are critically examined alongside emerging strategies to enhance specificity. With ongoing clinical trials and advancements in delivery systems, CRISPR technology represents a paradigm shift in addressing HD and broader neurodegenerative conditions. This review underscores the promise of gene editing in overcoming existing barriers and paving the way for transformative therapeutic approaches.

Indexed as

CRISPR-Cas SystemsGene EditingGenetic TherapyHuntington DiseaseAnimalsDisease Models, AnimalHumansHuntingtin ProteinHuntingtin ProteinCRISPR-Cas9Gene therapyHTT geneHuntington's diseaseNeurodegenerative disorders

Identifiers

PMID41171314

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.