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Gene therapy
Human Organic Anion Transporting Polypeptide 1B3
Applied as an MRI-Based Reporter Gene
Article
Year: 2020
Pages: 10
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Pharmacological Approaches in Neurofibromatosis Type 1-Associated Nervous System Tumors
Article
Year: 2021
Pages: 1
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Therapeutic Potential of
EWSR1–FLI1
Inactivation by CRISPR/Cas9 in Ewing Sarcoma
Article
Year: 2021
Pages: 1
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AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
Article
Year: 2020
Pages: 15
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AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells
in vitro
and human liver tumors in a murine xenograft model
in vivo
Article
Year: 2020
Pages: 13
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Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases
Article
Year: 2021
Pages: 1
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Long-Term Decrease of Intraocular Pressure in Rats by Viral Delivery of miR-146a
Article
Year: 2021
Pages: 1
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Recent Advances in Stimulus‐Responsive Nanocarriers for Gene Therapy
Article
Year: 2021
Pages: 1
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CMIP interacts with WT1 and targets it on the proteasome degradation pathway
Article
Year: 2021
Pages: 1
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Lysosomal Storage Disorders: Molecular Basis and Therapeutic Approaches
Article
Year: 2021
Pages: 1
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Personalized Medicine in Parkinson’s Disease: New Options for Advanced Treatments
Article
Year: 2021
Pages: 1
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How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?
Article
Year: 2021
Pages: 1
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Albumin Nanostructures for Nucleic Acid Delivery in Cancer: Current Trend, Emerging Issues, and Possible Solutions
Article
Year: 2021
Pages: 1
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Gene Therapy in Hemophilia: Recent Advances
Article
Year: 2021
Pages: 1
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Evaluation and Optimization of Poly-
d
-Lysine as a Non-Natural Cationic Polypeptide for Gene Transfer in Neuroblastoma Cells
Article
Year: 2021
Pages: 1
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Drug Delivery via the Suprachoroidal Space for the Treatment of Retinal Diseases
Article
Year: 2021
Pages: 1
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New Perspectives of Gene Therapy on Polyglutamine Spinocerebellar Ataxias: From Molecular Targets to Novel Nanovectors
Article
Year: 2021
Pages: 1
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Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives
Article
Year: 2021
Pages: 1
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On the Relationship of Viral Particles and Extracellular Vesicles: Implications for Viral Vector Technology
Article
Year: 2021
Pages: 1
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Mesenchymal Stem/Stromal Cell-Based Delivery: A Rapidly Evolving Strategy for Cancer Therapy
Article
Year: 2021
Pages: 1
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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 Gene Networks Underlying Retinal Recovery
Article
Year: 2021
Pages: 15
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Exploiting Single-Cell Tools in Gene and Cell Therapy
Article
Year: 2021
Pages: 1
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Molekulare Medizin: Pathobiochemie als Schlüssel zur personalisierten Therapie vererbter Krankheiten
Article
Year: 2021
Pages: 9
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Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa
Article
Year: 2021
Pages: 23
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National guidelines for gene therapy product (2019): A road-map to gene therapy products development and clinical trials
Article
Year: 2021
Pages: 8
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Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine
Article
Year: 2021
Pages: 1
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Angiogenesis and nerve regeneration induced by local administration of plasmid
pBud-coVEGF165-coFGF2
into the intact rat sciatic nerve
Article
Year: 2021
Pages: 8
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The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery
Article
Year: 2021
Pages: 9
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Potential of Mitochondrial Genome Editing for Human Fertility Health
Article
Year: 2021
Pages: 1
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Lentiviral and AAV-mediated expression of palivizumab offer protection against Respiratory Syncytial Virus infection
Article
Year: 2021
Pages: 1
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Functionalized DMP-039 Hybrid Nanoparticle as a Novel mRNA Vector for Efficient Cancer Suicide Gene Therapy
Article
Year: 2021
Pages: 22
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Substantial restoration of night vision in adult mice with congenital stationary night blindness
Article
Year: 2021
Pages: 11
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Innovative dual system approach for selective eradication of cancer cells using viral-based delivery of natural bacterial toxin–antitoxin system
Article
Year: 2021
Pages: 13
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Disrupting the LINC complex by AAV mediated gene transduction prevents progression of Lamin induced cardiomyopathy
Article
Year: 2021
Pages: 1
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Tweaking Progranulin Expression: Therapeutic Avenues and Opportunities
Article
Year: 2021
Pages: 1
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Endocardial fibroelastosis and dilated cardiomyopathy – the past and future of the interface between histology and genetics
Article
Year: 2021
Pages: 7
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AAV ablates neurogenesis in the adult murine hippocampus
Article
Pages: 1
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Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Article
Year: 2021
Pages: 1
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PAMAM-Functionalized Cellulose Nanocrystals with Needle-Like Morphology for Effective Cancer Treatment
Article
Year: 2021
Pages: 1
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Potential of helper-dependent Adenoviral vectors in CRISPR-cas9-mediated lung gene therapy
Article
Year: 2021
Pages: 1
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Pharmacogenetic information in Swiss drug labels – a systematic analysis
Article
Year: 2020
Pages: 12
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The Present and Future of Mitochondrial-Based Therapeutics for Eye Disease
Article
Year: 2021
Pages: 1
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Highly efficient gene editing and single cell analysis of hematopoietic stem/progenitor cells from X-linked sideroblastic anemia patients
Article
Year: 2021
Pages: 1
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Mesenchymal stem cells as professional actors in gastrointestinal cancer therapy: From Naïve to genetically modified
Article
Pages: 16
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Primary caregiver decision-making in hematopoietic cell transplantation and gene therapy for sickle cell disease
Article
Year: 2020
Pages: 1
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A systematic review of economic evaluations of advanced therapy medicinal products
Article
Year: 2020
Pages: 16
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Cardiac Overexpression of XIN Prevents Dilated Cardiomyopathy Caused by
TNNT2
ΔK210 Mutation
Article
Year: 2021
Pages: 1
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Defining inclusion criteria and endpoints for clinical trials: a prospective cross‐sectional study in
CRB1
‐associated retinal dystrophies
Article
Year: 2021
Pages: 13
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Effective intraperitoneal gene transfection system using nanobubbles and ultrasound irradiation
Article
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Room for improvement in the treatment of pancreatic cancer: Novel opportunities from gene targeted therapy
Article
Year: 2021
Pages: 13
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Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia
Article
Year: 2021
Pages: 10
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Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1
Article
Year: 2021
Pages: 10
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Modular Lentiviral Vectors for Highly Efficient Transgene Expression in Resting Immune Cells
Article
Year: 2021
Pages: 1
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Mesenchymal Stem Cells Engineered by Nonviral Vectors: A Powerful Tool in Cancer Gene Therapy
Article
Year: 2021
Pages: 1
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Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta‐analysis of preclinical studies
Article
Year: 2021
Pages: 12
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Novel Therapeutic Advances in β-Thalassemia
Article
Year: 2021
Pages: 1
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Engineered EV-Mimetic Nanoparticles as Therapeutic Delivery Vehicles for High-Grade Serous Ovarian Cancer
Article
Year: 2021
Pages: 1
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Therapy Prospects for Mitochondrial DNA Maintenance Disorders
Article
Year: 2021
Pages: 1
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Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases
Article
Year: 2021
Pages: 1
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Transduction Enhancers Enable Efficient Human Adenovirus Type 5-Mediated Gene Transfer into Human Multipotent Mesenchymal Stromal Cells
Article
Year: 2021
Pages: 1
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Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
Article
Year: 2021
Pages: 1
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Riboswitches for Controlled Expression of Therapeutic Transgenes Delivered by Adeno-Associated Viral Vectors
Article
Year: 2021
Pages: 1
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Erythropoietin Gene Therapy Delays Retinal Degeneration Resulting from Oxidative Stress in the Retinal Pigment Epithelium
Article
Year: 2021
Pages: 1
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Effect of Inducible BMP-7 Expression on the Osteogenic Differentiation of Human Dental Pulp Stem Cells
Article
Year: 2021
Pages: 1
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Mesenchymal Stem Cells as a Gene Delivery Tool: Promise, Problems, and Prospects
Article
Year: 2021
Pages: 1
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Planet of the AAVs: The Spinal Cord Injury Episode
Article
Year: 2021
Pages: 1
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Surface Modification of
E. coli
Outer Membrane Vesicles with Glycosylphosphatidylinositol-Anchored Proteins: Generating Pro/Eukaryote Chimera Constructs
Article
Year: 2021
Pages: 1
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Considerations in the Design of Non-Clinical Development Programmes to Support Non-Viral Genetically Modified Mesenchymal Stromal Cell Therapies
Article
Year: 2021
Pages: 1
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Engineering Genetic Systems for Treating Mitochondrial Diseases
Article
Year: 2021
Pages: 1
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New Therapies to Correct the Cystic Fibrosis Basic Defect
Article
Year: 2021
Pages: 1
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α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice
Article
Year: 2021
Pages: 1
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AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia
Article
Year: 2021
Pages: 1
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Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells
Article
Year: 2021
Pages: 22
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Transfer of a human gene variant associated with exceptional longevity improves cardiac function in obese type 2 diabetic mice through induction of the SDF‐1/CXCR4 signalling pathway
Article
Year: 2020
Pages: 14
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Double VEGF/HGF Gene Therapy in Critical Limb Ischemia Complicated by Diabetes Mellitus
Article
Year: 2020
Pages: 7
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The targeted inhibition of prostate cancer by iron-based nanoparticles based on bioinformatics
Article
Year: 2020
Pages: 12
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Calcium enhances polyplex-mediated transfection efficiency of plasmid DNA in Jurkat cells
Article
Pages: 11
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Synergistic ultrasonic biophysical effect-responsive nanoparticles for enhanced gene delivery to ovarian cancer stem cells
Article
Pages: 16
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CRISPR Pioneers Win 2020 Nobel Prize for Chemistry
Article
Pages: 5
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Spatiotemporal control of CRISPR/Cas9 gene editing
Article
Year: 2021
Pages: 1
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Poly(beta-amino ester) nanoparticles enable tumor-specific TRAIL secretion and a bystander effect to treat liver cancer
Article
Year: 2021
Pages: 12
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Biological and Medical Applications of Calcium Phosphate Nanoparticles
Article
Year: 2021
Pages: 18
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RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model
Article
Year: 2020
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Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
Article
Pages: 1
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Is gene therapy for limb ischemia a reality?
Article
Pages: 1
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Fe-doped chrysotile nanotubes containing siRNAs to silence
SPAG5
to treat bladder cancer
Article
Year: 2021
Pages: 1
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An Overview of RNA-Based Scaffolds for Osteogenesis
Article
Year: 2021
Pages: 1
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Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
Article
Year: 2021
Pages: 1
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The Role of Physiotherapy in the New Treatment Landscape for Haemophilia
Article
Year: 2021
Pages: 1
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Therapeutic Perspectives of Thermogenic Adipocytes in Obesity and Related Complications
Article
Year: 2021
Pages: 1
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DMD/BMD prenatal diagnosis and treatment expectation in a single centre in China for 15 years
Article
Year: 2021
Pages: 1
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Phototoxicities Caused by Continuous Light Exposure Were Not Induced in Retinal Ganglion Cells Transduced by an Optogenetic Gene
Article
Year: 2021
Pages: 1
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Inhibition of Hepatitis B Virus by AAV8-Derived CRISPR/SaCas9 Expressed From Liver-Specific Promoters
Article
Year: 2021
Pages: 1
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CRISPReader System Sensing the Ets-1 Transcription Factor Can Effectively Identify Cancer Cells
Article
Year: 2021
Pages: 1
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Congenital Deafness and Recent Advances Towards Restoring Hearing Loss
Article
Year: 2021
Pages: 1
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Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes
Article
Year: 2021
Pages: 18
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Autosomal recessive osteopetrosis: mechanisms and treatments
Article
Year: 2021
Pages: 1
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Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
Article
Year: 2021
Pages: 12
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Adeno-associated viral (AAV) vector-mediated therapeutics for diabetic cardiomyopathy – current and future perspectives
Article
Year: 2021
Pages: 19
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Leber Hereditary Optic Neuropathy: Review of Treatment and Management
Article
Year: 2021
Pages: 1
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3’ UTR-truncated HMGA2 overexpression induces non-malignant
in vivo
expansion of hematopoietic stem cells in non-human primates
Article
Year: 2021
Pages: 9
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Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A
ND4
Mutation: Systematic Review and Indirect Comparison
Article
Year: 2021
Pages: 1
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Intramuscular Expression of Plasmid-Encoded FVII-Fc Immunoconjugate for Tumor Immunotherapy by Targeting Tumoral Blood Vessels and Cells
Article
Year: 2021
Pages: 1
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Genome-based therapeutic interventions for β-type hemoglobinopathies
Article
Year: 2021
Pages: 1
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Suppression of lysosomal acid alpha‐glucosidase impacts the modulation of transcription factor EB translocation in pancreatic cancer
Article
Year: 2021
Pages: 14
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RPE65
-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy
Article
Year: 2021
Pages: 1
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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice
Article
Year: 2021
Pages: 1
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Overexpression of human BAG3
P209L
in mice causes restrictive cardiomyopathy
Article
Year: 2021
Pages: 1
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Transplantation of Apoptosis‐Resistant Endothelial Progenitor Cells Improves Renal Function in Diabetic Kidney Disease
Article
Year: 2021
Pages: 1
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Challenging Safety and Efficacy of Retinal Gene Therapies by Retinogenesis
Article
Year: 2021
Pages: 1
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Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy
Article
Year: 2021
Pages: 1
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Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
Article
Year: 2021
Pages: 1
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Decoding LncRNAs
Article
Year: 2021
Pages: 1
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Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia
Article
Year: 2021
Pages: 1
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Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies
Article
Year: 2021
Pages: 1
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Use of high-content imaging to quantify transduction of AAV-PHP viruses in the brain following systemic delivery
Article
Year: 2021
Pages: 1
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Targeting the
Apoa1
locus for liver-directed gene therapy
Article
Year: 2021
Pages: 14
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2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia
Article
Year: 2021
Pages: 1
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Effect of CpG Depletion of Vector Genome on CD8
+
T Cell Responses in AAV Gene Therapy
Article
Year: 2021
Pages: 1
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Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo
Article
Year: 2021
Pages: 1
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Hematopoietic Stem Cell Therapy for Wiskott–Aldrich Syndrome: Improved Outcome and Quality of Life
Article
Year: 2021
Pages: 13
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Photostable and Biocompatible Fluorescent Silicon Nanoparticles for Imaging-Guided Co-Delivery of siRNA and Doxorubicin to Drug-Resistant Cancer Cells
Article
Year: 2019
Pages: 1
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Oral Delivery of Biologics in Inflammatory Bowel Disease Treatment
Article
Year: 2021
Pages: 1
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CRISPR/Cas based gene editing: marking a new era in medical science
Article
Year: 2021
Pages: 17
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Recent Advances in the Treatment of Hemophilia: A Review
Article
Year: 2021
Pages: 15
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Distinct expression requirements and rescue strategies for
BEST1
loss- and gain-of-function mutations
Article
Pages: 1
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Challenges Posed by Immune Responses to AAV Vectors: Addressing Root Causes
Article
Year: 2021
Pages: 1
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Single-Dose Lentiviral Mediated Gene Therapy Recovers CFTR Function in Cystic Fibrosis Knockout Rats
Article
Year: 2021
Pages: 1
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Systemic cell therapy for muscular dystrophies
The ultimate transplantable muscle progenitor cell and current challenges for clinical efficacy
Article
Year: 2020
Pages: 22
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A Review of Acquired Autoimmune Blistering Diseases in Inherited Epidermolysis Bullosa: Implications for the Future of Gene Therapy
Article
Year: 2021
Pages: 1
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Next Step in Gene Delivery: Modern Approaches and Further Perspectives of AAV Tropism Modification
Article
Year: 2021
Pages: 1
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A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs)
Article
Year: 2021
Pages: 1
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Viral-mediated transduction of auditory neurons with opsins for optical and hybrid activation
Article
Year: 2021
Pages: 1
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Role of Biomolecules in Osteoclasts and Their Therapeutic Potential for Osteoporosis
Article
Year: 2021
Pages: 1
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Minicircles for Investigating and Treating Arthritic Diseases
Article
Year: 2021
Pages: 1
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Nucleic Acid Delivery with Red-Blood-Cell-Based Carriers
Article
Year: 2021
Pages: 1
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Renal Ciliopathies: Sorting Out Therapeutic Approaches for Nephronophthisis
Article
Year: 2021
Pages: 1
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Viral Gene Therapy for Glioblastoma Multiforme: A Promising Hope for the Current Dilemma
Article
Year: 2021
Pages: 1
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Surrogate Cerebrospinal Fluid Biomarkers for Assessing the Efficacy of Gene Therapy in Hurler Syndrome
Article
Year: 2021
Pages: 1
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Design and Validation of a Process Based on Cationic Niosomes for Gene Delivery into Novel Urine-Derived Mesenchymal Stem Cells
Article
Year: 2021
Pages: 1
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Contemporary Transposon Tools: A Review and Guide through Mechanisms and Applications of
Sleeping Beauty
,
piggyBac
and
Tol2
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