ReviewHuman gene therapy2018
Gene Delivery to Joints by Intra-Articular Injection.
Review in Human gene therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06415461 (A Phase 1, Open Label Dose-Ranging Study to Assess the Safety, Tolerability, Preliminary Efficacy, and Dose Effect of CFL001 Cord Blood Product in Patients With Symptomatic Sacroiliac Joint Syndrome), which is not on this map. Cited by 78 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.
A Phase 1, Open Label Dose-Ranging Study to Assess the Safety, Tolerability, Preliminary Efficacy, and Dose Effect of CFL001 Cord Blood Product in Patients With Symptomatic Sacroiliac Joint Syndrome
Who cites it
78 citing papers in PubMed, 126 citations in OpenAlex.
- Inflammation-Responsive Nanocolloidal Hydrogel Enables On-Demand Gene Activation for Osteoarthritis Therapy.Advanced healthcare materials · 2026Article
- Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.Pharmaceutics · 2026Article
- Biologic and regenerative strategies for osteoarthritis: advances in MSC, exosome, and PRP based therapies.Frontiers in pharmacology · 2026Review
- The evolving landscape of minimally invasive procedures in musculoskeletal diseases-part I.Frontiers in medicine · 2026Review
- Nature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- LOXL2 alleviates post-traumatic knee osteoarthritis and pain.Journal of orthopaedic translation · 2025Article
- Navigating the Hurdles of Intra-Articular AAV Gene Therapy.International journal of molecular sciences · 2025Review
- Osteoarthritis gene therapy: Expanding the scope of genetic therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Breaking Down Osteoarthritis: Exploring Inflammatory and Mechanical Signaling Pathways.Life (Basel, Switzerland) · 2025Review
- Novel regulation mechanism of histone methyltransferase SMYD5 in rheumatoid arthritis.Cellular & molecular biology letters · 2025Article
- Therapeutic Controlled Release Strategies for Human Osteoarthritis.Advanced healthcare materials · 2025Review
- Ferroptosis in osteoarthritis: metabolic reprogramming, immunometabolic crosstalk, and targeted intervention strategies.Frontiers in immunology · 2025Review
- Engineered self-regulating macrophages for targeted anti-inflammatory drug delivery.Arthritis research & therapy · 2024Article
- A Review of Current Approaches to Pain Management in Knee Osteoarthritis with a Focus on Italian Clinical Landscape.Journal of clinical medicine · 2024Review
- Tissue engineering and future directions in regenerative medicine for knee cartilage repair: a comprehensive review.Croatian medical journal · 2024Review
- Animal Models of Osteoarthritis: Updated Models and Outcome Measures 2016-2023.Regenerative engineering and translational medicine · 2024Article
- Engineered Self-Regulating Macrophages for Targeted Anti-inflammatory Drug Delivery.Research square · 2024Article
- Potential therapeutic strategies for osteoarthritis via CRISPR/Cas9 mediated gene editing.Reviews in endocrine & metabolic disorders · 2024Review
- Rationally engineered novel AAV capsids for intra-articular gene delivery.Molecular therapy. Methods & clinical development · 2024Article
- Streamlined, single-step non-viral CRISPR-Cas9 knockout strategy enhances gene editing efficiency in primary human chondrocyte populations.Arthritis research & therapy · 2024Article
18 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
3 authors at 3 institutions in 1 country.
Funding
Abstract
Most forms of arthritis are incurable, difficult to treat, and a major cause of disability in Western countries. Better local treatment of arthritis is impaired by the pharmacokinetics of the joint that make it very difficult to deliver drugs to joints at sustained, therapeutic concentrations. This is especially true of biologic drugs, such as proteins and RNA, many of which show great promise in preclinical studies. Gene transfer provides a strategy for overcoming this limitation. The basic concept is to deliver cDNAs encoding therapeutic products by direct intra-articular injection, leading to sustained, endogenous synthesis of the gene products within the joint. Proof of concept has been achieved for both in vivo and ex vivo gene delivery using a variety of vectors, genes, and cells in several different animal models. There have been a small number of clinical trials for rheumatoid arthritis (RA) and osteoarthritis (OA) using retrovirus vectors for ex vivo gene delivery and adeno-associated virus (AAV) for in vivo delivery. AAV is of particular interest because, unlike other viral vectors, it is able to penetrate deep within articular cartilage and transduce chondrocytes in situ. This property is of particular importance in OA, where changes in chondrocyte metabolism are thought to be fundamental to the pathophysiology of the disease. Authorities in Korea have recently approved the world's first arthritis gene therapy. This targets OA by the injection of allogeneic chondrocytes that have been transduced with a retrovirus carrying transforming growth factor-β
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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.