OOIR: Observatory of International Research

Papers

(The median citation count of Human Gene Therapy is 2. 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 2022-01-01 to 2026-01-01.)

Article	Citations
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	99
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	87
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	68
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	60
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	52
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	51
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	49
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	48
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	46
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	45
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	43
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	39
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	37
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	34
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	31
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	30
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	30
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	30
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	29
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	29
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	29
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	28
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	27
Abstracts	27
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	26

Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	26
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	25
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	25
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	23
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	23
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	23
Correction to: An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature, by Ramirez et al.	23
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	20
rAAV Production and Titration at the Microscale for High-Throughput Screening	20
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	20
SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis	20
Lived Experience with Gene Therapy	20
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	20
Development and Validation of a Liquid Chromatography-Tandem Mass Spectrometry Method for Sensitive Analysis of Residual Protein Tat Bh1–101 in Lentiviral Vectors for Gene Therapy	19
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy	19
Gene Therapy for Huntington's Disease: The Future Is in Gene Editing	18
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	17
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	17
Interview with Dr. Ronald Crystal	17
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	17
Conversion of the Liver into a Biofactory for DNaseI Using Adeno-Associated Virus Vector Gene Transfer Reduces Neutrophil Extracellular Traps in a Model of Systemic Lupus Erythematosus	17
Acknowledgment of Reviewers 2021	17
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	17
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	16
Genetic Modification of Limbs Using Ex Vivo Machine Perfusion	16
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	16
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	15
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	15
Progress in Respiratory Gene Therapy	15
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	15
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	15
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	15
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	14
Introduction to ESGCT 2024 Special Issue	14
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	14
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	14
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	14
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-	14
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	13
Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	13
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	13
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	13
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	13
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products	13
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	13
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	13
Liver Gene Therapy	12
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	12

Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	12
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	12
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	12
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	12
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	11
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	11
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	11
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	11
Bluebird Bio Eliminating 30% of Staff in Restructuring	11
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	11
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	11
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities	11
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	10
Hemophilia Gene Therapy: The End of the Beginning?	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	10
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	10
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8	10
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	10
The Future of Exon Skipping for Duchenne Muscular Dystrophy	9
Personalizing Oncolytic Virotherapy	9
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	9
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Rational Use of Immunosuppressive Corticosteroids in Liver-Directed Adeno-Associated Virus Gene Therapy Studies	9
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	9
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	9
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
Modulation of Immune Reaction in Hydrodynamic Gene Therapy for Hemophilia A	9
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	9
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	9
Development and Application of a Liquid Chromatography-Mass Spectrometry Method for Residual Iodixanol Quantification in AAV-Based Gene Therapy Product Development	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
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	8
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	8
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	8
Adenoviral Transduction of Dickkopf-1 Alleviates Silica-Induced Silicosis Development in Lungs of Mice	8
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys	8
Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	8
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations	8
Interview with Barry Byrne, MD/PhD	8
International Conference on Lymphocyte Engineering Author Index	8
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	8
HIV Tat-Conjugated Histone H3 Peptides Induce Tumor Cell Death Via Cellular Stress Responses	7
Gene Therapy for Immunoglobulin E, Complement-Mediated, and Eosinophilic Disorders	7
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration	7
Efficient LAMA1 Gene Activation by Epigenome Editing as a Therapeutic Approach for LAMA2-CMD	7
Akouos Therapy Restores 11-Year-Old Boy's Hearing After 1 Month	7
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	7
Medicinal Products Based on Adeno-Associated Viral Vectors: A Regulatory Perspective on the Potential Risk of Insertion-Mediated Tumorigenesis	7
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for Human Gene Therapy	7
Blood Cancers Reported in Seven Children Dosed with SKYSONA™	7
Intracisternal AAV9-MAG- hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice	7
Clustered Regularly Interspaced Short Palindromic Repeats and Clustered Regularly Interspaced Short Palindromic Repeats–Associated Protein 9 System: Factors Affecting Precision Gene Editing Efficiency	7
MPZL1 Promotes Lung Adenocarcinoma Progression by Enhancing Tumor Proliferation, Invasion, Migration, and Suppressing Immune Function via Transforming Growth Factor-β1	7
Biodistribution and Tolerability of AAV-PHP.B-CBh- SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles	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
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67 ^phox -Deficient Chronic Granulomatous Disease	7
Gene Therapy Restores Auditory Functions in an Adult Vglut3 Knockout Mouse Model	7
Targeting Oncolytic Adenoviruses to Cancer Cells Using a Designed Ankyrin Repeat Protein Lipocalin-2 Fusion Protein	7
FDA Investigating Sarepta’s Elevidys® after Second Patient Dies	7
Timely Intervention: Navigating Ethical Challenges in OTOF -Gene Therapy Trials	7
Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair	7
Stable and Predictable Lentiviral Vector Production at Clinical Scale	7
The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy	7
Prevalence of Pre-Existing Neutralizing Antibodies Against Adeno-Associated Virus Serotypes 1, 2, 5, 6, 8, and 9 in Sera of Different Pig Strains	6
International Conference on Lymphocyte Engineering Author Index	6
Interview with Dr. David Williams	6
Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A	6
A Review of the Legislation of Direct-to-Consumer Genetic Testing in China	6
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	6
Acknowledgment of Reviewers 2022	6
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	6
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies	6
USP7-SOX9-miR-96-5p-NLRP3 Network Regulates Myocardial Injury and Cardiomyocyte Pyroptosis in Sepsis	6
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression	6
Heart of the Matter: AAV8 Improves Cardiac Function	6
Suicide Gene Delivery System Mediated by Ultrasound-Targeted Microbubble Destruction: A Promising Strategy for Cancer Therapy	6

Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia	6
Call for Papers: Special Issue on Toxicity And Safety in Clinical AAV Gene Therapy	6
Acknowledgment of Reviewers 2023	6
Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction	6
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain	6
Remembrances of Kenneth Berns, PhD	6
The Legal Status and Improvement Path of Human Genetic Data in Gene Therapy in China	6
Modulating Oncolytic Adenovirus Immunotherapy by Driving Two Axes of the Immune System by Expressing 4-1BBL and CD40L	6
Remembrances of Nicholas Muzyczka, PhD	6
Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System	6
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression	6
Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells	5
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction	5
Bone Marrow Mesenchymal Stem Cell-Derived Exosomes microRNA-31-5p Repress Pulmonary Fibrosis via IGFBP7	5
Evaluation of Purification Methods for Minimizing Transgene Expression Background During Viral Manufacturing	5
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors	5
Improvement of Precision in Recombinant Adeno-Associated Virus Infectious Titer Assay with Droplet Digital PCR as an Endpoint Measurement	5
Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model	5
Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice	5
Hospital Exemption for Advanced Therapy Medicinal Products in Spain	5
Genetic Modification of the AAV5 Capsid with Lysine Residues Results in a Lung-Tropic Liver-Detargeted Gene Transfer Vector	5
Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR Reactivation	5
Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity	5
Efficacy and Safety of a Krabbe Disease Gene Therapy	5
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease	5
Vertex, Enlaza Launch Up-to-$2B Collaboration to Improve CASGEVY® Conditioning	5
Central Nervous System-Targeted Gene Therapy for the Treatment of Neurocognitive Deficits in Mucopolysaccharidosis Type II Mice	5
Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases	5
Autologous Production: The Future of Sustainable Antibody Treatments	5
Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors	5
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	4
Recombinant Adeno-Associated Virus Vector Mediated Gene Editing in Proliferating and Polarized Cultures of Human Airway Epithelial Cells	4
Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy	4
Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors	4
Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy	4
Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids	4
Food and Drug Administration Sets Stage for Approval of First Duchenne Muscular Dystrophy Gene Therapy	4
The Lived Experience of Pediatric Gene Therapy: A Scoping Review	4
Kallistatin Improves High-Fat-Induced Insulin Resistance via Epididymal Adipose Tissue-Derived Exosomes	4
Nonhuman Primate Adenoviruses of the Human Adenovirus B Species Are Potent and Broadly Acting Oncolytic Vector Candidates	4
Verve Pauses Enrollment in Base Editing Trial after Adverse Events	4
Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches	4
Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatment	4
Call for Special Issue Papers: Genetic and Nongenetic Inflammation Networks in Major Human Diseases	4
How Great a Risk Do You Take? A Qualitative Study Exploring Attitudes of Individuals with Friedreich Ataxia Toward Gene Therapy	4
Interview with Dr. Florian Eichler	4
Comparison of Physical Perturbation Devices for Enhancing Lentiviral Vector-Mediated Gene Transfer to the Airway Epithelium	4
Sarepta Pauses, then Resumes, Shipments of DMD Gene Therapy	4
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4	4
Precision BioSciences, Novartis Launch Up-to-$1.5B In Vivo Genome Editing Collaboration	4
Higher Transduction Efficiency of AAV5 to Neural Stem Cells and Immature Neurons in Gerbil Dentate Gyrus Compared to AAV2 and rh10	3
Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy	3
Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona Case	3
Higher Seroprevalence of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies Among Racial Minorities in the United States	3
Beam Results Show First Genetic Correction of Disease-Causing Mutation	3
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration	3
Preclinical Safety and Biodistribution in Mice Following Single-Dose Intramuscular Inoculation of Tumor DNA Vaccine by Electroporation	3
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys	3
Cyclosporin H Improves the Transduction of CD34 ⁺ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease	3
International Conference on Lymphocyte Engineering 12–14 September 2023 Munich, Germany	3
International Conference on Lymphocyte Engineering 31 March–2 April 2022 Munich, Germany	3
Interview with Beverly Davidson, PhD	3
CD19 or CD20 CAR T Cell Therapy Demonstrates Durable Antitumor Efficacy in Patients with Central Nervous System Lymphoma	3
Developing a Gene Therapy for the Treatment of Autosomal Dominant Alzheimer's Disease	3
Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases	3
Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans	3
Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1	3
Prevention of Portal-Tract Fibrosis in Zfyve19 ^−/− Mouse Model with Adeno-As	3
Genetic Engineering in CAR T Cells for Solid Tumors: Current State, Barriers and Future Developments	3
Cure Rare Disease: An Initiative to Enable N of 1 Gene Editing	3
Preferential Expansion of Human CD34 ⁺ CD133 ⁺ CD90 ⁺	3
Correction to: Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors, by Westhaus et al.	3
Focused Ultrasounds as an Adeno-Associated Virus Gene Therapy-Empowering Tool in Juvenile Mice via Intracerebroventricular Administration	3
Advances in Off-Target Detection for CRISPR-Based Genome Editing	3
Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome	3
Thrombotic Microangiopathy Associated with Systemic Adeno-Associated Virus Gene Transfer: Review of Reported Cases	3
Retraction of: HOXD-AS1 Exerts Oncogenic Functions and Promotes Chemoresistance in Cisplatin-Resistant Cervical Cancer Cells by Chi et al. (doi: 10.1089/hum.2017.256)	3
Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy	3
Patient Dies after Treatment with Intellia CRISPR Therapy in Phase III Trial	3
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A	3
Correction to: Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis, by Wu et al. Hum Gene Th	2
Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model an	2
Have a Little Heart (or Not): Highly Minimized Skeletal Muscle Regulatory Cassettes with Low or No Activity in the Heart	2
Alpharetroviral Vector–Mediated Gene Therapy for IL7RA-Deficient Severe Combined Immunodeficiency	2
Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma	2
After He Jiankui's Case: Chinese Legislative Modifications in Human Embryo Gene Editing	2
Deciphering Key Adenoviral Elements in the Production of Recombinant Adeno-Associated Virus Vectors	2
Advancements in Gene-Based Therapeutic Angiogenesis for Chronic Limb-Threatening Ischemia	2
Oncolytic Vaccinia Virus Encoding Aphrocallistes vastus Lectin Suppresses the Proliferation of Gastric Cancer Cells	2
Lipid Nanoparticle mRNA Therapy Improves Survival and Reduces Serum Branched-Chain Amino Acids in Mouse Models of Maple Syrup Urine Disease	2
10-Month-Old Boy Makes History as World’s First Patient Treated with Personalized CRISPR Therapy	2
The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases	2
Secretion of 4-1BB Ligand Crosslinked to PD-1 Checkpoint Inhibitor Potentiates Chimeric Antigen Receptor T Cell Solid Tumor Efficacy	2
Oncolytic Adenovirus Armoring with CXCL9 and IL15 Shows Potent Antitumor Activity and Boosts CAR-T Therapy	2
Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus	2