| Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN |
76 |
| Effective Targeting of Multiple B-Cell Maturation Antigen-Expressing Hematological Malignances by Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T Cells |
31 |
| Nucleofection with Plasmid DNA for CRISPR/Cas9-Mediated Inactivation of Programmed Cell Death Protein 1 in CD133-Specific CAR T Cells |
22 |
| MALAT1-Driven Inhibition of Wnt Signal Impedes Proliferation and Inflammation in Fibroblast-Like Synoviocytes Through CTNNB1 Promoter Methylation in Rheumatoid Arthritis |
18 |
| CAR T Cells in Trials: Recent Achievements and Challenges that Remain in the Production of Modified T Cells for Clinical Applications |
17 |
| An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4(-/-) Mice |
17 |
| LncRNA HOXA-AS2 Facilitates Tumorigenesis and Progression of Papillary Thyroid Cancer by Modulating the miR-15a-5p/HOXA3 Axis |
16 |
| AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome |
15 |
| Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque |
14 |
| Preclinical Efficacy and Safety of CD19CAR Cytokine-Induced Killer Cells Transfected with Sleeping Beauty Transposon for the Treatment of Acute Lymphoblastic Leukemia |
14 |
| Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals |
14 |
| In Vivo Ovarian Cancer Gene Therapy Using CRISPR-Cas9 |
14 |
| A Phase 1 Trial of Oncolytic Adenovirus ICOVIR-5 Administered Intravenously to Cutaneous and Uveal Melanoma Patients |
14 |
| Purification of mRNA Encoding Chimeric Antigen Receptor Is Critical for Generation of a Robust T-Cell Response |
14 |
| Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys |
13 |
| In vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency |
13 |
| Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy |
13 |
| Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction |
13 |
| Development of Anti-Human Mesothelin-Targeted Chimeric Antigen Receptor Messenger RNA-transfected Peripheral Blood Lymphocytes for Ovarian Cancer Therapy |
13 |
| Altered Long Non-Coding RNA Transcriptomic Profiles in Ischemic Stroke |
13 |
| Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease |
13 |
| Gene Delivery to Joints by Intra-Articular Injection |
12 |
| Preclinical Models in Chimeric Antigen Receptor-Engineered T-Cell Therapy |
12 |
| Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with no Detectable Immunity |
12 |
| Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease |
12 |
| Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome Locus |
11 |
| CD33-Specific Chimeric Antigen Receptor T Cells with Different Co-Stimulators Showed Potent Anti-Leukemia Efficacy and Different Phenotype |
11 |
| Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion |
11 |
| STAT3-Activated Long Non-Coding RNA Lung Cancer Associated Transcript 1 Drives Cell Proliferation, Migration, and Invasion in Hepatoblastoma Through Regulation of the miR-301b/STAT3 Axis |
11 |
| Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients |
11 |
| Simultaneous Knockout of CXCR4 and CCR5 Genes in CD4+T Cells via CRISPR/Cas9 Confers Resistance to Both X4-and R5-Tropic Human Immunodeficiency Virus Type 1 Infection |
10 |
| Immunoengineering of the Vascular Endothelium to Silence MHC Expression During Normothermic Ex Vivo Lung Perfusion |
10 |
| Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A |
10 |
| Assessment of AAV Vector Tropisms for Mouse and Human Pluripotent Stem Cell-Derived RPE and Photoreceptor Cells |
9 |
| Transducing Airway Basal Cells with a Helper-Dependent Adenoviral Vector for Lung Gene Therapy |
9 |
| An Oncolytic Adenovirus Targeting Transforming Growth Factor beta Inhibits Protumorigenic Signals and Produces Immune Activation: A Novel Approach to Enhance Anti-PD-1 and Anti-CTLA-4 Therapy |
9 |
| AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease) |
9 |
| Hepatocyte Growth Factor Gene Therapy for Ischemic Diseases |
9 |
| Chimeric Antigen Receptors in Different Cell Types: New Vehicles Join the Race |
9 |
| Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice |
9 |
| Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease |
9 |
| Effect of Human Wnt10b Transgene Overexpression on Peri-Implant Osteogenesis in Ovariectomized Rats |
8 |
| Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy |
8 |
| Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis |
8 |
| Transplantation of Hepatocyte Growth Factor-Modified Dental Pulp Stem Cells Prevents Bone Loss in the Early Phase of Ovariectomy-Induced Osteoporosis |
8 |
| A Cetuximab-Mediated Suicide System in Chimeric Antigen Receptor-Modified Hematopoietic Stem Cells for Cancer Therapy |
8 |
| Gene Therapy in Lipoprotein Lipase Deficiency: Case Report on the First Patient Treated with Alipogene Tiparvovec Under Daily Practice Conditions |
8 |
| Interleukin-35 Gene-Modified Mesenchymal Stem Cells Protect Concanavalin A-Induced Fulminant Hepatitis by Decreasing the Interferon Gamma Level! |
8 |
| Oncolytic Viruses for Tumor Precision Imaging and Radiotherapy |
8 |
| Gene Therapy for Hemoglobinopathies |
8 |
