ReviewMolecular therapy. Methods & clinical development2016
Clinical development of gene therapy: results and lessons from recent successes.
Review in Molecular therapy. Methods & clinical development, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 111 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
111 citing papers in PubMed, 1 synthesis or guideline pooled it, 226 citations in OpenAlex.
- Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies.Gene therapy · 2023Pooled it
- Translational Barriers to AAV and CRISPR Gene Therapy in Diabetes.Saudi medical journal · 2026Review
- Harnessing State-of-the-Art Gene Therapy to Transform Oral Cancer Treatment.Biochemical genetics · 2026Review
- Bifidobacterium infantis-mediated HSV-TK/GCV therapy modulates the tumor microenvironment through site-specific phosphorylation of HIF-1α, mTOR, and PD-L1.Frontiers in oncology · 2026Article
- Combine mitochondrial-targeted gene therapy and chemotherapy to treat triple-negative breast cancer.Journal of experimental & clinical cancer research : CR · 2025Article
- Unlocking the potential: advancements and applications of gene therapy in severe disorders.Annals of medicine · 2025Review
- Glutathione hybrid poly (beta-amino ester)-plasmid nanoparticles for enhancing gene delivery and biosafety.Journal of advanced research · 2025Article
- Recent developments in translational imaging of in vivo gene therapy outcomes.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Preclinical evaluation of the efficacy and safety of adeno-associated virus 8-tissue-nonspecific alkaline phosphatase-D10 in Alpl-/- and AlplPrx1/Prx1 mouse models for the treatment of early and late-onset hypophosphatasia.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2025Article
- Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications.Journal of biomedical science · 2025Review
- CRISPR-Cas9 Gene Therapy: Non-Viral Delivery and Stimuli-Responsive Nanoformulations.Molecules (Basel, Switzerland) · 2025Review
- Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives.Current genomics · 2025Review
- Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy.Current gene therapy · 2025Review
- Physicochemical and biological impacts of light stress on adeno-associated virus serotype 6.Molecular therapy. Methods & clinical development · 2024Article
- mLumiOpto Is a Mitochondrial-Targeted Gene Therapy for Treating Cancer.Cancer research · 2024Article
- Therapeutic approaches targeting aging and cellular senescence in Huntington's disease.CNS neuroscience & therapeutics · 2024Review
- Gene therapy for ultrarare diseases: a geneticist's perspective.Journal of biomedical science · 2024Review
- Application of BMP-2 and its gene delivery vehicles in dentistry.The Saudi dental journal · 2024Review
- Exploration of Gene Therapy for Alport Syndrome.Biomedicines · 2024Review
- An Innovative Mitochondrial-targeted Gene Therapy for Cancer Treatment.bioRxiv : the preprint server for biology · 2024Article
51 more citing papers are in PubMed but not listed here.
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 at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Therapeutic gene transfer holds the promise of providing lasting therapies and even cures for diseases that were previously untreatable or for which only temporary or suboptimal treatments were available. For some time, clinical gene therapy was characterized by some impressive but rare examples of successes and also several setbacks. However, effective and long-lasting treatments are now being reported from gene therapy trials at an increasing pace. Positive outcomes have been documented for a wide range of genetic diseases (including hematological, immunological, ocular, and neurodegenerative and metabolic disorders) and several types of cancer. Examples include restoration of vision in blind patients, eradication of blood cancers for which all other treatments had failed, correction of hemoglobinopathies and coagulation factor deficiencies, and restoration of the immune system in children born with primary immune deficiency. To date, about 2,000 clinical trials for various diseases have occurred or are in progress, and many more are in the pipeline. Multiple clinical studies reported successful treatments of pediatric patients. Design of gene therapy vectors and their clinical development are advancing rapidly. This article reviews some of the major successes in clinical gene therapy of recent years.
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.