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 2020-11-01 to 2024-11-01.)

Article	Citations
Bone Marrow Mesenchymal Stem Cells-Derived Exosomal MicroRNA-150-3p Promotes Osteoblast Proliferation and Differentiation in Osteoporosis	61
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A	57
Choosing the Right Tool for Genetic Engineering: Clinical Lessons from Chimeric Antigen Receptor-T Cells	51
Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates	46
Fourth Boy Dies in Clinical Trial of Astellas' AT132	45
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy	43
Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity	42
Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles	39
Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma	39
Central Nervous System Therapeutic Targets in Friedreich Ataxia	34
Extracellular Vesicles Derived from Chimeric Antigen Receptor-T Cells: A Potential Therapy for Cancer	32
Retinal Tropism and Transduction of Adeno-Associated Virus Varies by Serotype and Route of Delivery (Intravitreal, Subretinal, or Suprachoroidal) in Rats	32
Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography	31
Efficacy and Safety of a Krabbe Disease Gene Therapy	31
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	29
Host Immune Responses after Suprachoroidal Delivery of AAV8 in Nonhuman Primate Eyes	29
CRISPR/Cas-Dependent and Nuclease-FreeIn VivoTherapeutic Gene Editing	28
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping	28
Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy	28
Capillary Electrophoresis-Sodium Dodecyl Sulfate with Laser-Induced Fluorescence Detection as a Highly Sensitive and Quality Control-Friendly Method for Monitoring Adeno-Associated Virus Capsid Protei	28
Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate	26
Genome-Editing Strategies for Treating Human Retinal Degenerations	26
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	26
Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates	26
Gene Editing for the Treatment of Primary Immunodeficiency Diseases	25

Adenosine Deaminase 1 Overexpression Enhances the Antitumor Efficacy of Chimeric Antigen Receptor-Engineered T Cells	25
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration	25
Is Hospital Exemption an Alternative or a Bridge to European Medicines Agency for Developing Academic Chimeric Antigen Receptor T-Cell in Europe? Our Experience with ARI-0001	24
The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System	24
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys	24
Clinical Course and Risk Factors of Disease Deterioration in Critically Ill Patients with COVID-19	23
Optimized Reversed-Phase Liquid Chromatography/Mass Spectrometry Methods for Intact Protein Analysis and Peptide Mapping of Adeno-Associated Virus Proteins	23
Optimized Adeno-Associated Virus Vectors for Efficient Transduction of Human Retinal Organoids	23
A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis	23
Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System	22
Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1	21
Long-Noncoding RNA TUG1 Promotes Parkinson's Disease via Modulating MiR-152-3p/PTEN Pathway	21
Combining Oncolytic Viruses with Chimeric Antigen Receptor T Cell Therapy	19
The AAV9 Variant Capsid AAV-F Mediates Widespread Transgene Expression in Nonhuman Primate Spinal Cord After Intrathecal Administration	19
The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer	19
Detailed Protocol for the Novel and Scalable Viral Vector Upstream Process for AAV Gene Therapy Manufacturing	19
Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy	19
Size Exclusion Chromatography with Dual Wavelength Detection as a Sensitive and Accurate Method for Determining the Empty and Full Capsids of Recombinant Adeno-Associated Viral Vectors	19
Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches	19
Genetic Rescue of X-Linked Retinoschisis Mouse (Rs1^−/y) Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8-RS1 Gene Transfer and Identifies	18
miR-375- and miR-1-Regulated Coxsackievirus B3 Has No Pancreas and Heart Toxicity But Strong Antitumor Efficiency in Colorectal Carcinomas	18
Neutralizing Antibody Evasion and Transduction with Purified Extracellular Vesicle-Enveloped Adeno-Associated Virus Vectors	18
Systematic Characterization of the Biodistribution of the Oncolytic Virus M1	18
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy	17
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression	17
HIV Gene Therapy: An Update	17
Safe and EffectiveIn VivoTargeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development	17
Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results	16
Oncolytic Adenovirus Type 3 Coding for CD40L Facilitates Dendritic Cell Therapy of Prostate Cancer in Humanized Mice and Patient Samples	15
Building on Synthetic Immunology and T Cell Engineering: A Brief Journey Through the History of Chimeric Antigen Receptors	15
BioMarin's ROCTAVIAN Wins Food and Drug Administration Approval As First Gene Therapy for Severe Hemophilia A	15
Prevalence of Adeno-Associated Virus 3 Capsid Binding and Neutralizing Antibodies in Healthy and Hemophilia B Individuals from India	15
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial	15
The Identification and Development of a Novel Oncolytic Virus: Alphavirus M1	14
CASGEVY Makes History as FDA Approves First CRISPR/Cas9 Genome Edited Therapy	14
Higher Seroprevalence of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies Among Racial Minorities in the United States	14
Liver Gene Therapy	14
Intravitreal Injection of an Exosome-Associated Adeno-Associated Viral Vector Enhances Retinoschisin 1 Gene Transduction in the Mouse Retina	14
Comprehensive Analysis of Cell Therapy on Chronic Skin Wound Healing: A Meta-Analysis	13
Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based Spacer	13
Progress in Respiratory Gene Therapy	13
Optimized Protocol for Accurate Titration of Adeno-Associated Virus Vectors	13
CD19 or CD20 CAR T Cell Therapy Demonstrates Durable Antitumor Efficacy in Patients with Central Nervous System Lymphoma	13
Hemophilia Gene Therapy: The End of the Beginning?	13
USP7-SOX9-miR-96-5p-NLRP3 Network Regulates Myocardial Injury and Cardiomyocyte Pyroptosis in Sepsis	12
Regulatory T Cells and Diabetes Mellitus	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies	12
Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes	12
Gene Transfer in Adeno-Associated Virus Seropositive Rhesus Macaques Following Rapamycin Treatment and Subcutaneous Delivery of AAV6, but Not Retargeted AAV6 Vectors	12

Induced Liver Regeneration Enhances CRISPR/Cas9-Mediated Gene Repair in Tyrosinemia Type 1	12
Chemical Modifications of the Capsid for Redirecting and Improving the Efficacy of Adeno-Associated Virus Vectors	12
Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1	11
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	11
Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors	11
Call for More Effective Regulation of Clinical Trials with Advanced Therapy Medicinal Products Consisting of or Containing Genetically Modified Organisms in the European Union	11
Real-Time Visualization and Quantification of Oncolytic M1 Virus In Vitro and In Vivo	11
Conditioning Regimens in Long-Term Pre-Clinical Studies to Support Development of Ex Vivo Gene Therapy: Review of Nonproliferative and Proliferative Changes	11
Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3^Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood	11
In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa	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
Food and Drug Administration Lifts Clinical Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Linked to Patient Death	11
Improved Potency and Safety of DNA-Encoded Antibody Therapeutics Through Plasmid Backbone and Expression Cassette Engineering	11
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	11
Adeno-Associated Virus-Mediated Gene Therapy for Patients' Fibroblasts, Induced Pluripotent Stem Cells, and a Mouse Model of Congenital Adrenal Hyperplasia	10
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors	10
Design and Assessment of Novel Anti-CD30 Chimeric Antigen Receptors with Human Antigen-Recognition Domains	10
Adeno-Associated Virus Delivery Limitations for Neurological Indications	10
Gene Therapy Restores Auditory Functions in an Adult Vglut3 Knockout Mouse Model	10
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities	10
Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy	10
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	10
CRISPR-Editing Therapy for Duchenne Muscular Dystrophy	10
Knockout-Induced Pluripotent Stem Cells for Disease and Therapy Modeling of IL-10-Associated Primary Immunodeficiencies	10
Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction	10
Characterization of Adeno-Associated Virus Capsid Proteins with Two Types of VP3-Related Components by Capillary Gel Electrophoresis and Mass Spectrometry	10
Comparison of Efficiency and Function of Vascular Endothelial Growth Factor Adenovirus Vectors in Endothelial Cells for Gene Therapy of Placental Insufficiency	10
Systemic scAAV9.U1a.hSGSH Delivery Corrects Brain Biochemistry in Mucopolysaccharidosis Type IIIA at Early and Later Stages of Disease	10
After Analysis, Bluebird Bio Says Vector “Very Unlikely” Cause of Acute Myeloid Leukemia	9
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	9
Towards Human Translation of Lentiviral Airway Gene Delivery for Cystic Fibrosis: A One-Month CFTR and Reporter Gene Study in Marmosets	9
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	9
The Future of Exon Skipping for Duchenne Muscular Dystrophy	9
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells	9
Sustained Oligomycin Sensitivity Conferring Protein Expression in Cardiomyocytes Protects Against Cardiac hypertrophy Induced by Pressure Overload via Improving Mitochondrial Function	9
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	9
Krüppel-Like Factor 15 Reduces Ischemia-Induced Apoptosis Involving Regulation of p38/MAPK Signaling	9
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	9
Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells	9
In Vivo Gene Therapy for Canine SCID-X1 Using Cocal-Pseudotyped Lentiviral Vector	9
Viral Vectors Expressing Interleukin 2 for Cancer Immunotherapy	9
Minimum Effective Dose to Achieve Biochemical Correction with Adeno-Associated Virus Vector-Mediated Gene Therapy in Mice with Pompe Disease	8
Ex VivoGene Delivery to Porcine Cardiac Allografts Using a Myocardial-Enhanced Adeno-Associated Viral Vector	8
Adeno-Associated Virus-Mediated Overexpression of Interleukin-10 Affects the Immunomodulatory Properties of Equine Bone Marrow-Derived Mesenchymal Stem Cells	8
M11: A Tropism-Modified Oncolytic Adenovirus Arming with a Tumor-Homing Peptide for Advanced Ovarian Cancer Therapies	8
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	8
Nonhuman Primate Adenoviruses of the Human Adenovirus B Species Are Potent and Broadly Acting Oncolytic Vector Candidates	8
Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors	8
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	8
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	8
Oncolytic Adenovirus ORCA-010 Activates Proinflammatory Myeloid Cells and Facilitates T Cell Recruitment and Activation by PD-1 Blockade in Melanoma	8
Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy	8
Sample Preparation Matters for Peptide Mapping to Evaluate Deamidation of Adeno-Associated Virus Capsid Proteins Using Liquid Chromatography–Tandem Mass Spectrometry	8
Insight and Development of Advanced Recombinant Adeno-Associated Virus Analysis Tools Exploiting Single-Particle Quantification by Multidimensional Droplet Digital PCR	8
Prevalence of Pre-Existing Neutralizing Antibodies Against Adeno-Associated Virus Serotypes 1, 2, 5, 6, 8, and 9 in Sera of Different Pig Strains	8
Bringing Gene Therapies for HIV Disease to Resource-Limited Parts of the World	8
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	7
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	7
Application of Size Exclusion Chromatography with Multiangle Light Scattering in the Analytical Development of a Preclinical Stage Gene Therapy Program	7
Oncolytic Herpes Simplex Virus Type 2 Can Effectively Inhibit Colorectal Cancer Liver Metastasis by Modulating the Immune Status in the Tumor Microenvironment and Inducing Specific Antitumor Immunity	7
Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis	7
Gene Therapy in Opn1mw^−/−/Opn1sw^−/− Mice and Implications for Blue Cone Monochromacy Patients with Deletion Mutations	7