ReviewMolecular therapy : the journal of the American Society of Gene Therapy2023
The state of cell and gene therapy in 2023.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 80 papers, 1 of them a synthesis that pooled 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.
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
80 citing papers in PubMed, 1 synthesis or guideline pooled it, 118 citations in OpenAlex.
- Outlook of Cell Gene Therapies Development and Approval from Quality and Regulatory Perspective.Therapeutic innovation & regulatory science · 2026Pooled it
- Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.Experimental & molecular medicine · 2026Article
- Anion-exchange chromatography separates structurally heterogeneous and low-potency particles in adeno-associated virus manufacture.Molecular therapy. Advances · 2026Article
- Liver-directed gene therapy results in amelioration of progressive familial intrahepatic cholestasis type 2 in mice.Hepatology communications · 2026Article
- Industry Perspective on Translational and Clinical Pharmacology Aspects of Viral-based Gene Therapies and Vaccines-Key Considerations and Learnings From Approved Products.The AAPS journal · 2026Review
- Genetically Modified MSCs for Targeted Regeneration: Balancing Efficacy, Biosafety, and GMP Standardization.Cells · 2026Review
- Proteasomal degradation of intracellularly expressed Amblyomin-X limits suicide gene therapy potential in melanoma cells.FEBS open bio · 2026Article
- Oncogenesis as an Adverse Effect of Gene Replacement Therapy in Hematopoietic Stem Cells.International journal of molecular sciences · 2026Review
- Comparative Transcriptomic Profiling Reveals Differences in Initiation of Antiviral Response in Low rAAV Producing HEK293 Suspension Cells.Biotechnology journal · 2026Article
- Spatiotemporal reprogramming of cardiac lipid metabolism by platelet-engineered RNA therapy epigenetically modulate heart repair and regeneration.Bioactive materials · 2026Article
- Extracellular vesicle-based delivery systems for nucleic acid therapeutics.Molecular therapy. Nucleic acids · 2026Review
- A single-cell multi-omics assay for simultaneous measurement of vector copy number and protein expression in CAR T cells.Molecular therapy. Advances · 2026Article
- Clinical pharmacology insights from recent cell and gene therapy approvals relevant to pediatrics.Pediatric research · 2026Review
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- Advancing training on genetic therapies: a multi-specialty survey highlighting the role of medical geneticists.European journal of human genetics : EJHG · 2026Article
- Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026Review
- Survey on new technologies and precision medicine to advance healthcare.Journal of community genetics · 2026Article
- Artificial Intelligence and the Transformation of Cell and Gene Therapy Development.Pharmaceutics · 2026Review
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery.Molecular therapy. Advances · 2026Article
20 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Progress in the understanding of human diseases has coincided with the advent of precision medicine, whereby the underlying genetic and molecular contributors can be used as diagnostic and therapeutic biomarkers. To address these, drug developers have designed a range of different treatment strategies, including gene therapy, which the American Society of Gene and Cell Therapy defines as the use of genetic material to treat or prevent disease. A number of approaches exist, including the delivery of genetic material in vivo or ex vivo, as well as the use of RNA species to alter gene expression in particular disease states. Through the end of the first quarter of 2023, there were more than 100 different approved gene, cell, and RNA therapies throughout the world, with over 3,700 more in clinical and preclinical development. This review comprehensively captures the landscape for such advanced therapies, including the different genetic technologies used and diseases targeted in clinical trials.
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.