OOIR: Observatory of International Research

Papers

(The TQCC of Human Gene Therapy is 6. 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 2021-02-01 to 2025-02-01.)

Article	Citations
Reuben Matalon, MD, PhD, FACMG (1935–2021)	61
Adeno-Associated Viral Vector-Delivered Pannexin-1 Mimetic Peptide Alleviates Airway Inflammation in an Allergen-Sensitized Mouse Model	59
Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System	57
Efficient Gene Transfer to Kidney Using a Lentiviral Vector Pseudotyped with Zika Virus Envelope Glycoprotein	57
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	45
Correction to: Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis, by Han et al. Hum Gene Ther 2024;35(3-4):9	43
Update: Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology by Juliette Hordeaux et al. Hum Gene Ther 2020;31:15–16. DOI: 10.1089/hum.2020.167	42
Heart of the Matter: AAV8 Improves Cardiac Function	40
Letter to the Editor: mRNA Vaccines in Urological Malignancies	39
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	34
Keys to Delivery: An Interview with Hildegard Büning	31
Correction to: Induced CD20 Expression on B-Cell Malignant Cells Heightened the Cytotoxic Activity of Chimeric Antigen Receptor Engineered T Cells, by Xu et al. Hum Gene Ther 2019;30(4):497–510	31
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	29
Viral Vectors Expressing Interleukin 2 for Cancer Immunotherapy	29
Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based Spacer	28
Correction to: Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice, by Xiao et al. Hum Gene Ther 2019;30(3):286–301; doi: 10.1089/hum.	28
ESGCTCollaborative Virtual CongressAbstract Author Index	28
Advances in Off-Target Detection for CRISPR-Based Genome Editing	28
A Review of the Legislation of Direct-to-Consumer Genetic Testing in China	26
Gene Supplementation in Mice Heterozygous for the D477G RPE65 Variant Implicated in Autosomal Dominant Retinitis Pigmentosa	26
Correction to: Stability and Safety of an AAV Vector for Treating RPGR-ORF15X-Linked Retinitis Pigmentosa by Deng, W.-T. et al. Hum Gene Ther 2015;26(9):593–602. DOI: 10.1089/hum.2015.03	25
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling	25
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”	25
Call for Papers: Special Issue on Toxicity And Safety in Clinical AAV Gene Therapy	24
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression	24

Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	24
Correction to: Generation of a Novel Oncolytic Vaccinia Virus Using the IHD-W Strain by Jaeil Shin et al. Hum Gene Ther 2021;32:9–10. DOI: 10.1089/hum.2020.050	24
Advances in MicroRNA Therapeutics: From Preclinical to Clinical Studies	23
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	23
Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element	23
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	21
Preclinical Safety and Biodistribution in Mice Following Single-Dose Intramuscular Inoculation of Tumor DNA Vaccine by Electroporation	20
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial	20
Characterization of Adeno-Associated Virus Capsid Proteins with Two Types of VP3-Related Components by Capillary Gel Electrophoresis and Mass Spectrometry	19
Peripheral Cellular Immune Responses Induced by Subretinal Adeno-Associated Virus Gene Transfer Can Be Restrained by the Subretinal-Associated Immune Inhibition Mechanism	19
Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy	19
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	19
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	19
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	19
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	19
Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy	18
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	18
Optimization of the Administration Strategy for the Armed Oncolytic Adenovirus ZD55-IL-24 in Both Immunocompromised and Immunocompetent Mouse Models	17
Acknowledgment of Reviewers 2023	17
M11: A Tropism-Modified Oncolytic Adenovirus Arming with a Tumor-Homing Peptide for Advanced Ovarian Cancer Therapies	17
ESGCT Abstract Author Index	16
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	15
Ocular Inflammation with Anti-Vascular Endothelial Growth Factor Treatments	15
Roche, Ascidian Launch Up-to-$1.8B RNA Exon Editing Collaboration	15
Restoring Ciliary Function: Gene Therapeutics for Primary Ciliary Dyskinesia	15
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	14
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	14
An Improved Lentiviral Fluorescent Genetic Barcoding Approach Distinguishes Hematopoietic Stem Cell Properties in MultiplexedIn VivoExperiments	14
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage	14
Therapeutic Efficacy of an Oncolytic Influenza Virus Carrying an Antibody Against Programmed Cell Death 1 in Hepatocellular Carcinoma	13
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	13
Development and Validation of an Anion Exchange High-Performance Liquid Chromatography Method for Analysis of Empty Capsids and Capsids Encapsidating Genetic Material in a Purified Preparation of Reco	13
Optimized Adeno-Associated Virus Vectors for Efficient Transduction of Human Retinal Organoids	13
Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death	13
Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors	13
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	13
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	12
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy	12
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors	12
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	12
Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome	12
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	12
Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System	12
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	11
Suicide Gene Delivery System Mediated by Ultrasound-Targeted Microbubble Destruction: A Promising Strategy for Cancer Therapy	11
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	11
Choosing the Right Tool for Genetic Engineering: Clinical Lessons from Chimeric Antigen Receptor-T Cells	11
Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction	11
Current Update on Severe Acute Respiratory Syndrome Coronavirus 2 Vaccine Development with a Special Emphasis on Gene Therapy Viral Vector Design and Construction for Vaccination	11

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	11
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	11
Innate Immune Sensing of Adeno-Associated Virus Vectors	11
Prevalence of Pre-Existing Neutralizing Antibodies Against Adeno-Associated Virus Serotypes 1, 2, 5, 6, 8, and 9 in Sera of Different Pig Strains	11
In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa	11
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	10
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction	10
AAV6-Mediated Gene Therapy Prevents Developmental Dentin Defects in a Dentinogenesis Imperfecta Type Ⅲ Mouse Model	10
Lentiviral Gene Therapy of Chronic Granulomatous Disease: Functional Assessment of Universal and Tissue-Specific Promoters	10
Clinical Course and Risk Factors of Disease Deterioration in Critically Ill Patients with COVID-19	10
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	10
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	10
Finnish Society of Gene and Cell Therapy: A Visionary and Creative Player in the Field	10
Biodistribution of Transplanted Hematopoietic Precursor Cells Injected Through Different Administration Routes in Newborn Mice	10
Bone Marrow Mesenchymal Stem Cell-Mediated Radiosensitive Promoter-Combined Sodium Iodide Symporter for the Treatment of Breast Cancer	10
The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer	10
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia	10
Review of Safety Data for Adenovirus 5 as a Delivery Vector for Intratumoral Cancer Gene Therapy	9
A Beautiful Mind and the Heart of an Explorer	9
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia	9
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	9
Treatment of a Hemophilia B Mouse Model with Platelet-Targeted Expression of Factor IX Padua	9
Biotechnological advances in gene therapy of hematopoietic stem cells: Systematic review and meta-analysis	9
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	9
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease	9
Remembrances of Nicholas Muzyczka, PhD	9
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	9
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	8
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients	8
Vector-Mediated Delivery of Human Major Histocompatibility Complex-I into Hepatocytes Enables Investigation of T Cell Receptor-Redirected Hepatitis B Virus-Specific T Cells in Mice, and in Macaque Cel	8
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	8
ESGCT Abstract Author Index	8
pUDK-HGF Gene Therapy to Relieve CLI Rest Pain and Ulcer: A Phase II, Double-Blind, Randomized Placebo-Controlled Trial	8
The RNA World: The Best of Times, the Worst of Times	8
Bluebird Bio Eliminating 30% of Staff in Restructuring	8
Nuclease-Deficient Clustered Regularly Interspaced Short Palindromic Repeat-Based Approaches for In Vitro and In Vivo Gene Activation	8
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	8
Gutless Helper-Dependent and First-Generation HAdV5 Vectors Have Similar Mechanical Properties and Common Transduction Mechanisms	8
Multimodular Optimization of Chemically Regulated T Cell Switches Demonstrates Flexible and Interchangeable Nature of Immune Cell Signaling Domains	8
Acknowledgment of Reviewers 2022	8
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis	8
Seroprevalence of Adeno-Associated Virus Neutralizing Antibodies in Males with Duchenne Muscular Dystrophy	8
Boy Dosed with Pfizer’s Duchenne Muscular Dystrophy Gene Therapy Dies a Year After Phase II Trial	8
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	8
Modulating Oncolytic Adenovirus Immunotherapy by Driving Two Axes of the Immune System by Expressing 4-1BBL and CD40L	7
Identification of Broadly Applicable Adeno-Associated Virus Vectors by Systematic Comparison of Commonly Used Capsid Variants In Vitro	7
Chemical Modifications of the Capsid for Redirecting and Improving the Efficacy of Adeno-Associated Virus Vectors	7
Strategies for Optimization of the Clustered Regularly Interspaced Short Palindromic Repeat-Based Genome Editing System for Enhanced Editing Specificity	7
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	6
Real-Time Visualization and Quantification of Oncolytic M1 Virus In Vitro and In Vivo	6
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	6
Short Time to Market and Forward Planning Will Enable Cell Therapies to Deliver R&D Pipeline Value	6
Genome-Editing Strategies for Treating Human Retinal Degenerations	6
Drug Repositioning for Amyloid Transthyretin Amyloidosis by Interactome Network Corrected by Graph Neural Networks and Transcriptome Analysis	6
The Identification and Development of a Novel Oncolytic Virus: Alphavirus M1	6
Optimized Protocol for Accurate Titration of Adeno-Associated Virus Vectors	6
Short-Term Steroid Treatment of Rhesus Macaque Increases Transduction	6
Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice	6
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	6
Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications	6
Call for Papers: Special Issue on Ocular Gene Therapy	6
Host Immune Responses after Suprachoroidal Delivery of AAV8 in Nonhuman Primate Eyes	6
Construction of Plasmid DNA Expressing Two Isoforms of Insulin-Like Growth Factor-1 and Its Effects on Skeletal Muscle Injury Models	6
Adeno-Associated Virus Vector-Mediated Expression of Antirespiratory Syncytial Virus Antibody Prevents Infection in Mouse Airways	6
miR-375- and miR-1-Regulated Coxsackievirus B3 Has No Pancreas and Heart Toxicity But Strong Antitumor Efficiency in Colorectal Carcinomas	6
In Vivo Hematopoietic Stem Cell Gene Therapy for SARS-CoV2 Infection Using a Decoy Receptor	6
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	6
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy	6
Hemophilia Gene Therapy: The End of the Beginning?	6
Efficacy and Safety of Dual-Targeting Chimeric Antigen Receptor-T Therapy for Relapsed or Refractory B Cell Lymphoid Malignancies: A Systematic Review and Meta-Analysis	6
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Exp	6
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	6
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	6
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	6
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	6
Combination Immunotherapy of Oncolytic Flu-Vectored Virus and Programmed Cell Death 1 Blockade Enhances Antitumor Activity in Hepatocellular Carcinoma	6
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	6