ReviewCureus2026
CRISPR-Cas9-Mediated Gene Editing in Hematological Disorders: Advancing Translational and Clinical Applications.
Review in Cureus, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9)-mediated gene editing has emerged as a pivotal advancement in hematological medicine, addressing genetic and regulatory abnormalities underlying inherited and acquired blood disorders. Conventional therapeutic strategies offer symptomatic control or partial disease modification, leaving substantial unmet clinical need for durable, mechanism-driven interventions. This narrative review aims to synthesize the translational and clinical progress of CRISPR-Cas9 applications in hematological disorders, with an emphasis on therapeutic strategies, safety considerations, and integration into clinical practice. A comprehensive literature evaluation was conducted using major biomedical databases, selecting peer-reviewed studies published between 2015 and 2025 that reported mechanistic, translational, or clinical relevance in hematology. Eligible literature included experimental investigations, clinical trials, and high-quality reviews, with exclusion of non-translational and germline-focused studies. This review highlights advances in hematopoietic stem cell editing, correction of hemoglobinopathies, immune cell engineering, and chimeric antigen receptor-based therapies, as well as progress in delivery platforms and high-fidelity nuclease designs. Key challenges related to off-target effects, genomic stability, manufacturing scalability, regulatory oversight, and ethical governance are critically evaluated. Emerging clinical trial outcomes demonstrate sustained therapeutic benefit and expanding feasibility across diverse hematological indications. Long-term clinical monitoring remains central to evaluating durability and safety. Collectively, current evidence supports CRISPR-Cas9 as a cornerstone technology in precision hematology, with continued optimization required to enable safe, equitable, and widespread clinical adoption.
Indexed as
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.