ArticleAdvanced pharmaceutical bulletin2013
Gene therapy, early promises, subsequent problems, and recent breakthroughs.
Article in Advanced pharmaceutical bulletin, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 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
36 citing papers in PubMed.
- Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.Genes · 2026Review
- Modern Approaches and Emerging Biological Therapies to Treat Fracture Nonunion.Pharmaceutics · 2025Review
- Lentiviral Vectors: From Wild-Type Viruses to Efficient Multi-Functional Delivery Vectors.International journal of molecular sciences · 2025Review
- Review
- Review
- Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy.Current gene therapy · 2025Review
- Establishment and Characterization of a Stable Producer Cell Line Generation Platform for the Manufacturing of Clinical-Grade Lentiviral Vectors.Biomedicines · 2024Article
- Review
- Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs.Communications biology · 2024Review
- Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy.Current gene therapy · 2024Review
- scAAV2-Mediated Expression of Thioredoxin 2 and C3 Transferase Prevents Retinal Ganglion Cell Death and Lowers Intraocular Pressure in a Mouse Model of Glaucoma.International journal of molecular sciences · 2023Article
- Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies.Pharmaceutics · 2023Review
- Electroactive nanoinjection platform for intracellular delivery and gene silencing.Journal of nanobiotechnology · 2023Article
- In the business of base editors: Evolution from bench to bedside.PLoS biology · 2023Article
- Targeting hormone-resistant breast cancer cells with docetaxel: a look inside the resistance.Cancer drug resistance (Alhambra, Calif.) · 2023Article
- The potential for mitochondrial therapeutics in the treatment of primary open-angle glaucoma: a review.Frontiers in physiology · 2023Review
- Nanoparticles-mediated CRISPR-Cas9 gene therapy in inherited retinal diseases: applications, challenges, and emerging opportunities.Journal of nanobiotechnology · 2022Review
- Combination of the modulators of epigenetic machinery and specific cell signaling pathways as a promising approach for cell reprogramming.Molecular and cellular biochemistry · 2022Review
- Digital Technologies: Advancing Individualized Treatments through Gene and Cell Therapies, Pharmacogenetics, and Disease Detection and Diagnostics.Biomedicines · 2022Article
- Obtaining a New Gene-Cell Construct Based on Transduced Olfactory Ensheathing Cells for the Treatment of Spinal Cord Injuries.Bulletin of experimental biology and medicine · 2022Article
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
Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to tissues for clinical applications has been discussed around half a century, but scientist's ability to manipulate genetic material via recombinant DNA technology made this purpose to reality. Various approaches, such as viral and non-viral vectors and physical methods, have been developed to make gene delivery safer and more efficient. While gene therapy initially conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, to date gene therapy is considered for many non-life-threatening conditions including those adversely influence on a patient's quality of life. Gene therapy has made significant progress, including tangible success, although much slower than was initially predicted. Although, gene therapies still at a fairly primitive stage, it is firmly science based. There is justifiable hope that with enhanced pathobiological understanding and biotechnological improvements, gene therapy will be a standard part of clinical practice within 20 years.
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.