ReviewMedComm2025
Gene therapy for genetic diseases: challenges and future directions.
Review in MedComm, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed.
- Next-Generation Bone Marrow Cell Therapies for Cardiac Repair: Integrating Gene Therapy and Bioengineering to Enhance Therapeutic Potency.Journal of cardiovascular translational research · 2026Review
- Self-Assembling Short Peptide Carriers for Gene Delivery.International journal of molecular sciences · 2026Review
- Ultrasound-guided left heart injection: a safer and more efficient strategy for mouse thoracic aortic gene delivery.Gene therapy · 2026Article
- Review
- Synaptic aging and neurodegeneration: the role of synaptic vesicle dynamics and neurotransmitter imbalance.Biogerontology · 2026Review
- Clinical translation of CRISPR-Cas9 therapeutics in cancer and inherited genetic disorders.Frontiers in genome editing · 2026Review
- Smart micro/nanoneedles for gene delivery.Iranian journal of basic medical sciences · 2026Review
- Genome editing between wonder and rejection.Frontiers in bioengineering and biotechnology · 2026Article
- Advances in Gene Therapy for X-Linked Myotubular Myopathy: Current Progress and Future Challenges.Current gene therapy · 2026Article
- Hybrid Nanocarriers for Cancer Therapy: Advancements in Co-Delivery of Gene Therapy and Immunotherapy.International journal of molecular sciences · 2025Review
- Advances in gene therapy for mitochondrial genetic disorders: current status and clinical implementation challenges.Journal of translational medicine · 2025Review
- Targeted Hepatic Delivery of Bioactive Molecules via Nanovesicles: Recent Developments and Emerging Directions.Journal of personalized medicine · 2025Review
- Beyond the Exome: The Role of Noncoding and Regulatory Variants in Monogenic Diseases.Current issues in molecular biology · 2025Review
- Generation of high-quality single-stranded DNA for full-length and truncated genome standards of recombinant adeno-associated viruses.Molecular therapy. Methods & clinical development · 2025Article
- Toward Precision Medicine: Gene Therapy Applications in the Management of Uveal Melanoma.Cancer reports (Hoboken, N.J.) · 2025Review
- Genomic medicine in hepatology: mechanisms and liver treatment strategies.Molecular medicine (Cambridge, Mass.) · 2025Review
- Article
- Novel Heterocyclic Compounds as SMN2 Modulators for Treating Spinal Muscular Atrophy Via Modulation of SMN2 Splicing.ACS medicinal chemistry letters · 2025Article
- Review
- A hierarchical and configurational analysis of Health Technology Assessment outcomes for cell and gene therapies.Frontiers in pharmacology · 2025Article
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
Genetic diseases constitute the majority of rare human diseases, resulting from abnormalities in an individual's genetic composition. Traditional treatments offer limited relief for these challenging conditions. In contrast, the rapid advancement of gene therapy presents significant advantages by directly addressing the underlying causes of genetic diseases, thereby providing the potential for precision treatment and the possibility of curing these disorders. This review aims to delineate the mechanisms and outcomes of current gene therapy approaches in clinical applications across various genetic diseases affecting different body systems. Additionally, genetic muscular disorders will be examined as a case study to investigate innovative strategies of novel therapeutic approaches, including gene replacement, gene suppression, gene supplementation, and gene editing, along with their associated advantages and limitations at both clinical and preclinical levels. Finally, this review emphasizes the existing challenges of gene therapy, such as vector packaging limitations, immunotoxicity, therapy specificity, and the subcellular localization and immunogenicity of therapeutic cargos, while discussing potential optimization directions for future research. Achieving delivery specificity, as well as long-term effectiveness and safety, will be crucial for the future development of gene therapies targeting genetic diseases.
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.