ReviewMolecular therapy. Nucleic acids2026
CRISPR-Cas9-mediated therapeutics: Current clinical trials and therapy approval landscape to treat human diseases.
Review in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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.
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.
Who cites it
5 citing papers in PubMed.
- Structural evolution of ionizable lipids for nucleic acid delivery.Nature reviews. Chemistry · 2026Review
- CRISPRing through time: How cutting-edge technology is revolutionizing life sciences and medicine.Molecular therapy. Nucleic acids · 2026Review
- Nanomedicine beyond carriers - devices, cells & living therapeutics.Biomedical microdevices · 2026Review
- Nanomaterials for the Prevention, Detection, and Treatment of Pharyngeal Human Papillomavirus Infection: A Translational Roadmap.Materials (Basel, Switzerland) · 2026Review
- Clinical translation of CRISPR-Cas9 therapeutics in cancer and inherited genetic disorders.Frontiers in genome editing · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The genome editing technology using CRISPR-Cas9 has created a new trajectory for treating diverse human diseases. The approval of CRISPR-Cas9 therapeutics was first given to Casgevy for treating transfusion-dependent β-thalassemia (TDT) by the UK Medicines and Healthcare Products Regulatory Agency (UKMHRA) on November 16, 2023. Subsequently, UKMHRA approved Casgevy for the treatment of sickle cell disease (SCD), which was approved by the US Food and Drug Administration (FDA) on December 8, 2023. The approval has created a new era of gene editing medicine. Numerous clinical trials have been initiated to treat different human diseases. In this comprehensive review, we present an overview of the therapeutic development that includes the earliest days of research using CRISPR-Cas9 technology, clinical trials, UKMHRA/FDA-approved therapeutics (Casgevy), and the CRISPR-Cas9 technology approved for the therapy of SCD and TDT. We also present details on the role of biopharmaceutical industry-academia collaboration and patent landscape of the CRISPR-Cas9 technology. Finally, the therapeutics' challenges, safety concerns, and cost-effectiveness are illustrated to provide a complete understanding on the current landscape of CRISPR-Cas9-mediated therapeutics for countries' policymakers, biopharmaceutical companies, and next-generation researchers to formulate the future strategies.
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Identifiers
What OpenQuestion holds
Registered trials
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.