OOIR: Observatory of International Research

Papers

(The TQCC of Human Gene Therapy is 7. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2022-07-01 to 2026-07-01.)

Article	Citations
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	94
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	57
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	47
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	47
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	44
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	41
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	35
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	34
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	34
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	33
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	32
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	32
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	31
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	29
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	29
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	26
Abstracts	26
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	24
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	23
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	23
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	22
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	22
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	22
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	21
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	20

Interview with Dr. Ronald Crystal	20
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	20
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	20
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	20
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	20
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	20
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	19
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	17
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	16
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	16
Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease Mice	15
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	15
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	15
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	14
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	14
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	13
Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-	13
Transduction of Ferret Surface and Basal Cells of Airways, Lung, Liver, and Pancreas via Intratracheal or Intravenous Delivery of Adeno-Associated Virus 1 or 6	13
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products	13
Introduction to ESGCT 2024 Special Issue	13
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	13
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12
Resolving the Unresolvable: Nanopore Sequencing as a Comprehensive Quality Control Platform for Gene Therapy Vectors	12
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	12
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	12
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	12
Progress in Respiratory Gene Therapy	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	11
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	11
Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	11
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	11
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	11
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	10
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	10
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	10
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	10
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	10
The Application of Cell and Gene-Modified Cell Therapy in the Treatment of Osteopetrosis	10
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	10
Retroviral Transduction of Human CD4 ⁺ T Cells with Membrane-Attached IL-10 Generates Type 1-Like Regulatory T Cells	10
The Future of Exon Skipping for Duchenne Muscular Dystrophy	10
Personalizing Oncolytic Virotherapy	10
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Stable and Predictable Lentiviral Vector Production at Clinical Scale	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
6 ^TH International Conference on Lymphocyte Engineering 15–17 July 2026 Milan, Italy	9
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	8

Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	8
Interview with Barry Byrne, MD/PhD	8
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	8
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67 ^phox -Deficient Chronic Granulomatous Disease	8
Correction to: Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatm	8
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys	8
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	8
HIV Tat-Conjugated Histone H3 Peptides Induce Tumor Cell Death Via Cellular Stress Responses	7
CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications	7
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	7
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	7
Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis	7
Intracisternal AAV9-MAG- hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice	7
Quantification of Linear Polyethylenimines in Recombinant Adeno-Associated Virus by High-Performance Liquid Chromatography with Charged Aerosol Detection	7
SG33, a Vaccine Strain of Myxoma Virus with Oncolytic Potential, Exploits Macropinocytosis and Clathrin-Mediated Endocytosis for Entry into Pancreatic Cancer Cells	7
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations	7
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for Human Gene Therapy	7
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration	7
A Review of the Challenge of Pre-Existing Humoral Immunity in Adeno-Associated Virus Gene Therapy and Potential Solutions	7
Medicinal Products Based on Adeno-Associated Viral Vectors: A Regulatory Perspective on the Potential Risk of Insertion-Mediated Tumorigenesis	7