ReviewFrontiers in genome editing2021
The Promise and the Hope of Gene Therapy.
Review in Frontiers in genome editing, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 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
32 citing papers in PubMed, 97 citations in OpenAlex.
- Anion-Exchange Membrane Chromatography for the Separation of Empty and Full Adeno-Associated Viral Capsids.Biotechnology journal · 2026Article
- Advancing Tuberculosis Chemotherapy: Targeted Nanomedicines for the Mycobacterium TB Granuloma.Small (Weinheim an der Bergstrasse, Germany) · 2025Review
- Tailored therapeutics for cardiomyopathies.Nature reviews. Cardiology · 2025Review
- Rational Design of Inner Ear Drug Delivery Systems.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Interlaboratory assessment of candidate reference materials for lentiviral vector copy number and integration site measurements.Molecular therapy. Methods & clinical development · 2025Article
- Recent developments in translational imaging of in vivo gene therapy outcomes.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- A Comprehensive Review of Current Approaches in Bladder Cancer Treatment.ACS pharmacology & translational science · 2025Review
- Navigating disruption in the PID landscape: embracing opportunities and anticipating threats in the next ten years.Frontiers in immunology · 2025Review
- Describing and Mapping the Research Trend of Scientific Publications on Arrhythmogenic Right Ventricular Cardiomyopathy Across Four Decades: A Bibliometric Analysis.Clinical cardiology · 2024Article
- An optimized protocol for quality control of gene therapy vectors using nanopore direct RNA sequencing.Genome research · 2024Article
- Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.Orphanet journal of rare diseases · 2024Article
- New epilepsy therapies in development.Nature reviews. Drug discovery · 2024Review
- The ABCs of Stargardt disease: the latest advances in precision medicine.Cell & bioscience · 2024Review
- Challenges for gene therapy in the financial sustainability of health systems: a scoping review.Orphanet journal of rare diseases · 2024Article
- Exploring the Therapeutic Potential of Gene Therapy in Arrhythmogenic Right Ventricular Cardiomyopathy.Biomedicines · 2024Review
- Evaluation of recombinant baculovirus clearance during rAAV production in Sf9 cells using a newly developed fluorescent-TCIDFrontiers in medicine · 2024Article
- Gene Therapy for Inherited Hearing Loss: Updates and Remaining Challenges.Audiology research · 2023Review
- Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies.Pharmaceutics · 2023Review
- Chitosan-Based Nanoparticles for Nucleic Acid Delivery: Technological Aspects, Applications, and Future Perspectives.Pharmaceutics · 2023Review
- Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies.International journal of molecular sciences · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
It has been over 30 years since visionary scientists came up with the term "Gene Therapy," suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
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.