ArticleMedical science monitor : international medical journal of experimental and clinical research2024
Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia.
Article in Medical science monitor : international medical journal of experimental and clinical research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 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
51 citing papers in PubMed.
- Molecular mechanisms and biotechnology applications of CRISPR-Cas12a.Nature reviews. Molecular cell biology · 2026Review
- SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.Gene therapy · 2026Article
- CRISPR integration of an FLT3 CAR into the TRAC locus generates CAR T cells for AML.Genome medicine · 2026Article
- Pre-existing antibody and T cell responses to SaCas9, AsCas12a and CasΦ are comparable in naïve individuals.Nature communications · 2026Article
- Integrating HTS and CRISPR/Cas for next-generation nucleic and non-nucleic acid diagnostics.Molecular genetics and genomics : MGG · 2026Review
- Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.Orphanet journal of rare diseases · 2026Article
- Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles.medRxiv : the preprint server for health sciences · 2026Article
- CRISPR-Cas9-mediated therapeutics: Current clinical trials and therapy approval landscape to treat human diseases.Molecular therapy. Nucleic acids · 2026Review
- Article
- Integrating natural and engineered genetic variations to decode regulatory influence on blood traits.Cell reports · 2026Article
- Gene modification: Exploring the potential in treating kidney diseases.Pharmacological research · 2026Review
- Sickle Cell Disease at a Tertiary Care Center in the Vidarbha Region of India: Protocol for a Clinical and Observational Study.JMIR research protocols · 2026Observational
- Gene, genetics and genetic medicines in gastroenterology: Current status and its future.World journal of gastroenterology · 2026Review
- CRISPR and Fanzor in sickle cell disease: current progress and future prospects.Frontiers in genome editing · 2026Review
- RNA therapeutic targeting of recalcitrant and rare cancers.Exploration of targeted anti-tumor therapy · 2026Review
- Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease.Cardiovascular & hematological disorders drug targets · 2026Review
- Enteric nervous system as a therapeutic target in gastrointestinal disorders.World journal of gastrointestinal pharmacology and therapeutics · 2025Article
- A xeno-free red blood cell differentiation formula models sickle cell disease from somatically sourced patient iPSCs.Experimental hematology · 2025Article
- Intraoperative CRISPR editing: the feasibility of gene surgery inside the operating room.Annals of medicine and surgery (2012) · 2025Article
- Stem cell revolution: bridging the gap between research and clinical application.Stem cell research & therapy · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In 2020, Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for their research on the endonuclease, clustered regularly interspaced short palindromic repeats (CRISPR) and the CRISPR-associated protein 9 (CRISPR-Cas9) method for DNA editing. On 16 November 2023, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) was the first to approve the CRISPR-Cas9 gene editing therapy, Casgevy (exagamglogene autotemcel), for the treatment of patients with transfusion-dependent b-thalassemia and the treatment of sickle cell disease in patients aged ≥12 years with recurrent vaso-occlusive crises. On 8 December 2023, the US Food and Drug Administration (FDA) approved both Casgevy and Lyfgenia (lovotibeglogene autotemcel) for patients with sickle cell disease. On 15 December 2023, the European Medicines Agency (EMA) approved Casgevy for sickle cell disease and transfusion-dependent ß-thalassemia. This Editorial aims to present an update on the landmark first regulatory approvals of CRISPR-Cas9 for patients with sickle cell disease and transfusion-dependent b-thalassemia and the potential challenges for therapeutic gene (DNA) editing.
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.